DCRT news

RARE-NL AON meeting @LUMC March 11

Thu, 05 Feb 2026 09:51:40 GMT

RARE-NL AON meeting @LUMC March 11

When and where
Wednesday 11 March 2026, 9.00-16.30
LUMC building 3, Buruma lecture hall

Morning program 9.00-13.30

From Model to Therapy, hDMT/RARE-NL Preclinical Network
This meeting is specifically aimed at preclinical researchers who use in vitro and in vivo preclinical disease models to develop therapy strategies for rare diseases. Regulators and other stakeholders in this area are also welcome to join.
Program and Registration
From Model to Therapy: RARE-NL/hDMT Preclinical Network - RARE-NL

Afternoon program 13.30-16.30

RARE-NL @LUMC – Antisense oligonucleotides for n-of-few: on the way to the clinic

This RARE-NL meeting focuses on the development and implementation of antisense oligonucleotides for n-of-few and n-of-1 indications. The meeting brings together researchers, clinicians and experts in access pathways to explore how ASOs can be translated into clinical practice. The emphasis is on shared challenges and solutions that require collaboration beyond individual UMCs.
Program and Registration
RARE-NL @LUMC - Antisense oligonucleotides for n-of-few: on the way to the clinic - RARE-NL

EMA webinar on Platform considerations for RNA therapies

Thu, 05 Feb 2026 09:29:03 GMT

Webinar on the use of platform technologies in the non-clinical and clinical domains

Webinar on the use of platform technologies in the non-clinical and clinical domains | European Medicines Agency (EMA)

Happy new year from all of us at DCRT

Mon, 05 Jan 2026 11:50:56 GMT

Happy new year from all of us at DCRT

posted on January 5, 2026

Roan Hofstede joins DCRT for an internship

Mon, 05 Jan 2026 11:46:20 GMT

Student Roan Hofstede joins DCRT for an internship

Hello all! I am Roan Hofstede and I have joined the DCRT team as an intern for 9-10 months as a part of my Bachelor's degree in Biology & Medical Laboratory Research at the Leiden University of Applied Sciences.

I will be working on a way to predict toxicity of intrathecally administered ASOs in a short-term study in rats, together with Raúl.

posted on January 5, 2026

Second DCRT newsletter

Wed, 12 Nov 2025 10:29:20 GMT

Second DCRT newsletter

Here is DCRT's second newsletter with information about new DCRT partner UMC Utrecht.

posted on November 12, 2025

PhD student Raul Andres Santamaria presented his research at the OTS in Budapest

Wed, 12 Nov 2025 10:11:41 GMT

PhD student Raul Andres Santamaria presented his research at the OTS in Budapest

DCRT participated in the 21st annual meeting of the Oligonucleotide Therapeutics Society in Budapest last month. Rob Collin gave a talk about his research in the field of rare eye diseases and both Willeke van Roon-Mom and Raul Andres Santamaria had prepared a poster presentation.

posted on November 12, 2025

DCRT's Rob Collin gave a presentation at the annual meeting of the OTS

Mon, 20 Oct 2025 14:09:03 GMT

DCRT's Rob Collin gave a presentation at the annual meeting of the OTS

Today, dr. Rob Collin, professor at Radboud University Medical Center and partner at DCRT, gave a presentation on splicing modulation therapy for inherited retinal diseases at the annual meeting of the Oligonucleotide Therapeutics Society in Budapest.

DCRT's Annemieke Aartsma-Rus, Alex Garanto and Emilio Harris-Mostert are members of the organizing committee of this year's meeting. Willeke van Roon-Mom and Raul Andres Santamaria will represent the DCRT at the poster sessions.

posted on October 20, 2025

DCRT at the annual meeting of the N=1 Collaborative in Budapest

Mon, 20 Oct 2025 13:40:48 GMT

DCRT at the annual meeting of the N=1 Collaborative

Prior to the OTS, the N=1 Collaborative gathered for their second annual meeting in Budapest.

DCRT was there too! Can you find how many DCRT people are in the picture?

posted on October 20, 2025

student Kamala Vermeer joins the DCRT for an internship

Thu, 02 Oct 2025 09:42:38 GMT

Student Kamala Vermeer joins the DCRT for an internship

Hello there! I am Kamala Vermeer and I have joined the DCRT team as an intern for 9 months as a part of my Bachelor's degree in Biology & Medical Laboratory Research at the Leiden University of Applied Sciences.

I will be working on the neurotoxicity pipeline and convert iPSCs to iAstrocytes together with Anouk & Marlen.

posted on October 2, 2025

DCRT's Marlen Lauffer at the American Society of Human Genetics Meeting in Boston

Mon, 22 Sep 2025 10:17:17 GMT

DCRT's Marlen Lauffer will give a workshop at the American Society of Human Genetics Meeting in Boston

Dr. Marlen Lauffer will give an interactive workshop on assessing genetic variants for ASO therapy at the American Society of Human Genetics Meeting in Boston on October 14th. Moreover, she will moderate Session 55 'Gene Editing and Gene Replacement for Rare Disease' on Thursday, October 16.

Marlen will be happy to meet with you and tell you more about DCRT's work on ASO therapy for very rare brain and eye diseases.

posted on September 22, 2025

UMC Utrecht and UMC Groningen to become DCRT partners

Wed, 13 Aug 2025 09:42:00 GMT

UMC Utrecht and UMC Groningen to become DCRT partners

We are pleased to announce that UMC Utrecht (Jan Veldink and Wouter van Rheenen, Department of Neurology) and UMC Groningen (Peter van den Akker and Jeroen Bremer, Department of Clinical Genetics) will become full member of the DCRT as per January 1 ^st , 2026. UMCU will bring expertise in rare variants of ALS and UMCG aims to develop ASO therapy for rare, genetic skin disorders.

posted on August 13, 2025

DCRT researchers to attend the N1C meeting and the OTS in Budapest

Thu, 31 Jul 2025 10:24:27 GMT

DCRT researchers to attend the N1C annual meeting and the OTS in Budapest in October

The DCRT will be in Budapest for the annual meeting of the N=1 Collaborative on 18 October and for the OTS annual meeting on 19-22 October 2025 (early bird fee valid up to September 19).

We hope to see you there!

posted on July 31, 2025

DCRT researchers attend the Dutch Antisense Therapeutics Symposium

Fri, 13 Jun 2025 12:09:44 GMT

DCRT presentations at the 4th DATS in Groningen

Wonderful to see that our DCRT researchers and students are doing such a great job presenting their work on the development of antisense oligonucleotides for ultra rare genetic diseases of the brain or the eye. With poster presentations by Raul, Marlen, Okke, Jack, Noortje, Anouk, Tracy, Bianca, Willeke and Nadine, and selected short talks by Edwin, Laurie, Redmar and Emilio, DCRT was very well represented at the DATS.

As in previous years, DCRT organized a dedicated session at the DATS, which took place this morning, and featured presentations by invited speakers Roxana Redis (Charles River) and Helene Tran (Servier) and by PhD students Seppe Hermans (BianoGMP) and Edwin van Oosten (ErasmusMC).

Internship for bachelor student Noortje Groenendijk

Wed, 07 May 2025 11:23:22 GMT

Bachelor student Noortje Groenendijk joins DCRT for an internship

Noortje is a Bachelor student at the DCRT within the LUMC. Her project involves the development of personalized antisense oligonucleotides (ASOs) for a rare neurodegenerative disorder called Leigh syndrome, caused by a mutation in the NDUFAF6 gene. This internship will be the final part of her Bachelor’s degree in Biomedical Sciences at the University of Leiden.

posted on May 7, 2025

DATS poster with speakers

Mon, 28 Apr 2025 09:09:06 GMT

Keynotes, invited speakers and sponsors at DATS 2025 in Groningen

All keynote and invited speakers have confirmed their participation in the Dutch Antisense Therapeutics Symposium on 12 and 13 June 2025 in Groningen! The deadline for early-bird registration and abstract submission (May 9th) is approaching fast. Don't forget to fill out the registration form on the website: www.dutchantisense.org . We are looking forward to seeing you in Groningen!

Master student Tracy Wüsthoff joins the DCRT

Thu, 20 Mar 2025 12:49:00 GMT

Master student Tracy Wüsthoff joins the DCRT for an internship

Tracy is a master student at the University of Potsdam, pursuing a degree in Biochemistry and Molecular Biology. She is currently conducting her thesis at the DCRT, where her project focuses on the development of allele-selective antisense oligonucleotides (ASOs) to target a heterozygous variant in ACTL6B, which is linked to an intellectual development disorder. The aim of her research is to specifically inactivate the mutated allele while ensuring that the wild-type allele remains functional, as individuals carrying a heterozygous recessive mutation in ACTL6B do not exhibit symptoms.

Tracy completed her bachelor degree in Biochemistry at Freie Universität Berlin before continuing her master studies at the University of Potsdam.

posted on March 20, 2025

Come join the DCRT at the Dutch Antisense Therapeutics Symposium in Groningen!

Wed, 19 Mar 2025 11:12:47 GMT

Come and join the DCRT at the Dutch Antisense Therapeutics Symposium in Groningen!

The 4th Dutch Antisense Therapeutics Symposium will be held on 12 and 13 June 2025 in Groningen.

There will be slots available for short talks by early career researchers. Abstract deadline (also for posters) is May 9th. The DCRT will organize a session on N=1 ASO therapy with invited speakers Roxana Redis (CR) and Helene Tran (Servier).

Registration is open! -> www.dutchantisense.org

posted on March 19, 2025

Master student Okke Faber joins the DCRT

Mon, 17 Mar 2025 10:32:17 GMT

Master student Okke Faber joins the DCRT

Okke Faber is a Master student at the DCRT. His project aims to develop a web-accessible database of genes containing protein tandem repeats, providing a resource for identifying and prioritizing potential therapeutic targets for antisense oligonucleotide (ASO) mediated exon skipping in missense variants. Okke is currently pursuing a Master degree in Molecular Genetics and Biotechnology at Leiden University, where he also obtained a Bachelor degree in Biology with a thesis on antibiotics resistance.

posted on March 17, 2025

Ype Elgersma will be interviewed on television about his work on epilepsy

Mon, 20 Jan 2025 13:22:33 GMT

DCRT's Ype Elgersma will be interviewed on television about his work on epilepsy

This Thursday, 23 January, at 17.10, Prof. Ype Elgersma (ErasmusMC) will talk on Dutch television about his research into RNA therapy for children with epilepsy. The television program is called 'Tijd voor MAX' and is broadcasted on NPO1.

posted on January 20, 2025

Happy Christmas holidays from the DCRT

Thu, 19 Dec 2024 11:46:40 GMT

Happy Christmas holidays from all of us at DCRT!

We wish you a lovely Christmas time with family and friends!

posted on December 19, 2024

Guest researchers at DCRT

Wed, 18 Dec 2024 10:18:29 GMT

Guest researchers at the DCRT

In the past month, two guest researchers visited DCRT. Dr. Julian W. März (photo on the left), a research fellow at the University of Zurich, has a keen interest in patent law, regulatory affairs & research ethics, with a focus on innovative medicines for rare diseases. He gave two lectures: Personalised Medicine: Need for a Paradigm Shift in European Patent Law and Public Funding for Individualized Therapeutics: A Review

of (Potential) Models in Europe. Seppe Hermans works at Biano GmbH in Germany and is a PhD student in the European doctoral network Medicine Made to Measure. His secondment at DCRT was aimed at learning about ASO design and development through interaction with his fellow MMM doctoral researchers and DCRT staff.

posted on December 18, 2024

4th Dutch Antisense Therapeutics Symposium in Groningen

duda-wsm@mijndomein.nl — Thu, 05 Dec 2024 14:36:32 GMT

Join us on 12 and 13 June 2025 in Groningen, The Netherlands, for the 4th Dutch Antisense Therapeutics Symposium

The 4th edition of the DATS will take place on 12 and 13 June 2025 at UMC Groningen, The Netherlands. DATS aims to strengthen oligonucleotide research between academia and industry in the Netherlands (and beyond). DATS offers a safe and friendly environment for early career researchers to present their work and to interact with peers, invited speakers and sponsors, also at the social event.
Of course, the DCRT will be there as well with a dedicated session on the development of oligonucleotide therapy for N=1 genetic diseases of the brain, the spinal cord and the eye.

posted on December 5, 2024

DCRT interview in FAST newsletter

Thu, 05 Dec 2024 13:55:03 GMT

DCRT interview in the newsletter of Centre for Future Affordable Sustainable Therapy Development (FAST)

In its recent newsletter , FAST published an interview with Annemieke Aartsma-Rus and Willeke van Roon-Mom about DCRT's efforts to develop tailored treatment with AONs for patients with a unique genetic disease. Dr Jasper Claessen from the Ministry of Health, Welfare and Sport welcomes this academic initiative but at the same time realizes that issues such as measurement of effectiveness and safety of the drug and the costs of personalized treatment must be addressed first.

posted on December 5, 2024

DCRT travel grant recipients

Mon, 04 Nov 2024 15:04:54 GMT

DCRT travel grant recipients, Edwin van Oosten and Raúl Andrés about the OTS meeting

The OTS meeting in Montreal was a nice experience for all PhD students. Here is a short impression from the DCRT travel grant recipients!

posted on November 4, 2024

First DCRT newsletter

Mon, 04 Nov 2024 13:00:16 GMT

First DCRT newsletter

This is the first newsletter of the DCRT. We plan to issue our newsletter two times per year: on April 1 st and November 1 st

posted on November 4th, 2024

Poster award for three DCRT colleagues

Thu, 24 Oct 2024 09:37:11 GMT

Marlen Lauffer, Emilio Harris-Mostert and Edwin van Oosten received an OTS poster award

At the annual meeting of the Oligonucleotide Therapeutics Society in Montreal, Marlen Lauffer, Emilio Harris-Mostert and Edwin van Oosten received an OTS poster award. Well done!

posted on October 31, 2024

DCRT's project manager celebrates 12.5 years of employment at LUMC

Thu, 24 Oct 2024 09:28:18 GMT

Cake and candles for 12.5 years at LUMC

DCRT's project manager, Pauline de Graaf, celebrated 12.5 years employment at the LUMC. Her colleagues bought cake and flowers today to celebrate!

Annemieke was presented with the Jacobs Ladder Award

Thu, 24 Oct 2024 09:14:02 GMT

Annemieke Aartsma-Rus was presented with the Norman Saunders Jacob's Ladder Award

Congratulations to Annemieke Aartsma-Rus for receiving the Norman Saunders Jacob’s Ladder Award at Sick Kids hospital in Toronto. This award is given to persons who have done excellent translational research in the neurology field. Annemieke is the 21 ^st recipient of this award, that is named after the late Jacob Schwarz, a patient with Canavan Disease, and Prof Norman Saunders, the clinician who managed the care for Jacob and helped the Schwarz family set up the Jacob’s Ladder Foundation.

Seminar Annemieke Aartsma_Rus at SickKids Research Institute and Jacob’s Ladder The Canadian Foundation for Control of Neurodegenerative Disease

Wed, 16 Oct 2024 08:26:54 GMT

Today, Annemieke Aartsma-Rus will receive the Norman Saunders Jacob's Ladder International Research Prize at the SickKids Research Institute in Canada

We are proud to announce that Annemieke Aartsma-Rus will be presented with the Norman Saunders Jacob's Ladder International Research Prize! Congratulations from all of us at the DCRT, Annemieke!

Prior to the award ceremony, Annemieke will give a seminar entitled 'How to bring individualized antisense oligonucleotides from bench to eligible patients?'

The seminar is today at 15.00 C.E.T. and can be followed via Zoom. Registration can be done via the zoom link in the announcement.

posted on October 16, 2024

DCRT at the Inaugural Annual Meeting of the N=1 Collaborative

Thu, 10 Oct 2024 13:06:55 GMT

Presentations by DCRT at the Inaugural Annual Meeting of N=1 Collaborative

Prior to the OTS meeting in Montreal, N=1 Collaborative held its first annual meeting. Jack Morgan, Willeke van Roon-Mom, Annemieke Aartsma-Rus and Marlen Lauffer presented on behalf of the DCRT.

Thanks to Imperfect JOY for the wonderful photos!

posted on October 10, 2024

DCRT colleagues together at the OTS meeting in Montreal

Thu, 10 Oct 2024 10:50:15 GMT

DCRT colleagues together at the OTS meeting in Montreal

DCRT colleagues attended the 20th Annual Meeting of the Oligonucleotide Therapeutics Society in Montreal. Annemieke Aartsma-Rus gave the keynote presentation on ASO-mediated exon skipping for individualized treatment whereas others presented a poster. At the end of the meeting, they gathered for a DCRT group photo!

posted on October 10,2024

DCRT at the OTS in Montreal

Wed, 09 Oct 2024 12:45:17 GMT

DCRT at the OTS in Montreal

Many DCRT colleagues are at the 20th Annual Meeting of the Oligonucleotide Therapeutics Society in Montreal, Canada, this week. You may have encountered them already! Here are the first two photos that made their way to the Netherlands: Raul Andres and Jack Morgan with their poster.

posted on October 9, 2024

Two new DCRT papers have been published online

Thu, 26 Sep 2024 12:52:52 GMT

Two new DCRT papers have been published online

Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics

Annemieke Aartsma-Rus, Rob W.J. Collin, Ype Elgersma, Marlen C. Lauffer, Willeke van Roon-Mom, 2024 (sagepub.com)

ExonViz: A website and Python package to visualize transcripts and genetic variants

Redmar R. van den Berg, Marlen C. Lauffer, Jeroen F.J. Laros

medRxiv 2024.09.18.24313945; doi: https://doi.org/10.1101/2024.09.18.24313945

The first paper is a joint effort by DCRT partners LUMC, Radboudumc and ErasmusMC and describes the work that the DCRT has been doing towards the development of an individualized ASO treatment for patients with an ultra-rare genetic, progressive disorder of the brain or the eye. The second paper describes the tool that Team LUMC has developed to visualize genetic transcripts and variants. Feel free to try out the ExonViz tool on our website: Exonviz (rnatherapy.nl) .

posted on September 26, 2024

Team DCRT at Escape Rooms in Leiden

Thu, 08 Aug 2024 13:16:53 GMT

Team DCRT went for its annual outing to Leiden's escape rooms

While Willeke, Annemieke, Marlen, Redmar and Pauline (photo) were trying to find Rembrandt's golden paint brush to be able to escape from Leiden's wall tower 'Oistenrijck', the second group successfully escaped from 'Wonderland' wearing silly hats, hence no photo permission.... After our adventures, we assembled for drinks at the Burcht, a medieval stronghold in the historic center of Leiden, dating back to the 11th century!

posted on August 8, 2024

N=1 Collaborative is looking for a communications trainee

Thu, 08 Aug 2024 09:11:06 GMT

N=1 Collaborative is looking for communication trainees

The N=1 Collaborative is a nonprofit whose mission is to transform the treatment of rare genetic disease by building a scalable and responsible pathway for individualized medicines. We are a team of clinicians, scientists, regulators, patient advocates from across academia, government, foundations, and biotech/pharma committed to openly sharing and iterating our work with the broader community in order to make individualized medicines routine worldwide.

N1C is looking for volunteers to help with science communication, social media and website management, and generation and curation of internal and public resources.

For more information, please contact info@n1collaborative.org.

posted on August 8, 2024

PhD position for 3 years at the Center for Neurology in Tuebingen

Wed, 07 Aug 2024 12:15:59 GMT

PhD Position in treatment-response fluid biomarkers for ASO treatments

The Doctoral Training Network “Medicine Made to Measure” (MMM) is looking for a PhD student for the Translational Genomics of Neurodegenerative Diseases division at the University of Tübingen, Germany.

The PhD candidate will be supported for 3 years by the European Union Horizon Marie Sklodowska Curie Actions programme. The successful applicant will attend regular training courses with other MMM PhD students and will be given the opportunity to gain experience at MMM partner institutes, such as Leiden University Medical Center.

If you are interested, carefully read the job description and send your application before 18th August to s

selina.reich@uni-tuebingen.de.

posted on August 7, 2024

DCRT at the Dutch Antisense Therapeutics Symposium

Wed, 07 Aug 2024 11:52:49 GMT

Short report of the 3rd Dutch Antisense Therapeutics Symposium in Rotterdam

The third DATS was held on 23 and 24 May 2024 at the Arminius church in Rotterdam.

The programme consisted of keynote lectures, invited lectures and short talks as well as 28! poster presentations and a dedicated session organized by the DCRT. Keynote speakers were Shalini Andersson from AstraZeneca Sweden and Rob Collin from DCRT/Radboudumc.

For the DCRT session we had invited Floor Nieuwenhuijse and Bart van de Warrenburg (Radboudumc) and Matthis Synofzik (Tübingen University) to give a presentation. Annemieke and Willeke presented a DCRT update. Furthermore, there were DCRT short talks by PhD students Emilio, Laurie, Bianca and Edwin as well as a large number of DCRT posters.

The networking event took place at the city brewery where we had bites and beers and a pub quiz presented by Alex Garanto. At the end of the symposium, prizes were awarded for best short talk (Remko Goossens, runner-up Edwin van Oosten) and for best poster (Jack Morgan).

The next DATS will be held in Groningen on 12 and 13 June 2025!

posted on August 7, 2024

DCRT travel grants awarded

Wed, 31 Jul 2024 10:05:29 GMT

DCRT travel grants awarded to 3 PhD students

The DCRT board selected 3 PhD students, working on ASO development for N=1, to receive a travel grant to attend this year’s meeting of the Oligonucleotide Therapeutics Society in Montréal. We are pleased to announce that we have awarded the grants to:

Raúl Andrés Santamaria (LUMC)
Edwin van Oosten (Radboudumc)
Emilio Harris-Mostert (ErasmusMC)

Congratulations and enjoy the conference!

posted on July 31, 2024

Marlen Lauffer at the Genomics England research summit in London

Wed, 10 Jul 2024 11:21:00 GMT

Marlen Lauffer at the Genomics England Research Summit 2024

DCRT's Marlen Lauffer was invited to give a talk at the Genomics England Research Summit in London on the 9th of July. Her presentation covered DCRT's work on the identification of genes and diseases that would be eligible for our individualized RNA therapy as well as our recommendations for the selection of eligible patients. Photo courtesy of Haiyan Zhou.

posted on July 10, 2024

New DCRT paper has been published in Human Mutation

Wed, 01 May 2024 11:19:14 GMT

Our paper on patient selection guidelines for ASO-based treatments has been published in Human Mutation!

We are happy to announce that our paper entitled 'Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments' has been published in Human Mutation. Here is the link: https://www.hindawi.com/journals/humu/2024/9920230/ .

posted on May 1, 2024

Congratulations to Bianca for her article on KMT2

Mon, 22 Apr 2024 12:06:35 GMT

A special cake for Bianca Zardetto to celebrate the acceptance of her article in Human Mutation

Congratulations to Bianca! Her article on treatability of KMT2 -associated neurodevelopmental disorders using ASO therapy has been accepted for publication in Human Mutation. To celebrate the occasion, Willeke had a special cake made for her which was shared with the entire team ��.

posted on April 18, 2024

Dutch Antisense Therapeutics Symposium

Wed, 10 Apr 2024 11:46:44 GMT

DCRT at the 3rd Dutch Antisense Therapeutics Symposium in Rotterdam

In 6 weeks time, the third DATS will kick off in Rotterdam. The DCRT session with invited speakers Prof Matthis Synofzik and Prof Bart van de Warrenburg will take place on Friday, 24th May, 2024. More information on registration, preliminary program and abstract deadlines is available on the DATS website: www.dutchantisense.org.

posted on April 10, 2024

Student Nick van Dullemen joins the DCRT

Thu, 04 Apr 2024 08:00:44 GMT

Student Nick van Dullemen joins the DCRT for an internship

My name is Nick van Dullemen, a 3rd-year bachelor’s student in biomedical sciences at Leiden University. During my four-month internship at the DCRT, I will be working on evaluating the allele-specificity of an antisense oligonucleotide (ASO) targeting a specific mutation in a disease-causing gene. This project aims to see if the used ASOs inactivate the disease-causing mutated allele, while leaving the wild-type allele intact. Eventually, if the ASOs turn out to be allele-specific they could possibly be used to decrease the disease burden in the future. During my internship, I will mostly be focused on performing transfections, RNA extraction and several types of PCR to test the allele-specificity of the ASOs.

Welcome to the DCRT, Nick!

posted on March 14, 2024

Erasmus+ student Janina Lösch joins the DCRT for an internship

Wed, 27 Mar 2024 12:49:56 GMT

Erasmus+ student Janina Lösch joins the DCRT for an internship

Janina Lösch is a master student from University of Bonn doing an Erasmus+ internship at the DCRT. Her project focuses on allele selective ASOs targeting a heterozygous variant in ACTL6B that causes an intellectual development disorder. The idea is to inactivate the mutated allele while leaving the wild type allele intact, as heterozygous carriers of recessive mutations in ACTL6B are healthy.
Janina obtained her bachelor’s degree in Molecular Biomedicine after which she continued with a master’s program in Immunobiology, both at the University of Bonn. For her master thesis she investigated the effect of the menstrual cycle on blood immune cells.

Welcome to the DCRT, Janina!

posted on March 27, 2024

The CCMO (Central committee on research involving human subjects) celebrates its 25th anniversary on 5 April 2024

Mon, 04 Mar 2024 13:57:57 GMT

DCRT's Willeke van Roon-Mom presents on antisense oligonucleotides at the CCMO symposium

The CCMO (Central Committee on research involving human subjects) celebrates its 25th anniversary on the 5th of April 2024 with a scientific symposium on ethics and medicine. DCRT's Willeke van Roon-Mom is one of the speakers!

Read more about the program and registration on CCMO's LinkedIn page.

posted on March 4, 2024

It's Rare Disease Day and DCRT is celebrating her 4th birthday!

Thu, 29 Feb 2024 11:14:15 GMT

It's Rare Disease Day and DCRT is celebrating her 4th birthday!

The Dutch Center for RNA Therapeutics was founded on Rare Disease Day 2020 so it has been 4 years already that we have started our path towards developing therapies for ultra-rare genetic diseases of the brain, the spinal cord and the eye. We still learn every day about the rare diseases that exist in the world and we hope that our ASO therapy will one day make a huge difference in the life of someone suffering from a rare disease.

Happy birthday, DCRT!

posted on February 29, 2024

DCRT session at the third Dutch Antisense Therapeutics Symposium

Mon, 12 Feb 2024 14:09:13 GMT

DCRT session at the third Dutch Antisense Therapeutics Symposium

The third edition of the Dutch Antisense Therapeutics Symposium will be held on 23 and 24 May 2024 in Rotterdam. DCRT's Rob Collin has been invited as keynote speaker along with Dr. Shalini Andersson from AstraZeneca Sweden. The DCRT will organize a session about clinical developments in rare disease therapy. Furthermore, there are plenty of slots available for short talks and poster presentations by early career researchers and a great networking event on the 23rd. So go the the DATS website and register for this interesting event!

posted on February 12, 2024

Merry Christmas

Thu, 21 Dec 2023 14:16:51 GMT

Merry Christmas from all of us at DCRT

Happy holidays and until next year!

Marlen Lauffer highlighted in the N1C newsletter

Thu, 21 Dec 2023 12:01:27 GMT

Dr. Marlen Lauffer is highlighted member of this month's N1C newsletter

DCRT's Marlen Lauffer is highlighted member of the month in the N1C newsletter ! She is the lead of the N1C patient identification workgroup and, as such, working on a project to assess variants for ASO amenability.

Dream of the Dutch Center for RNA Therapeutics in 'Best Idea' of the Netherlands and Flanders

Mon, 18 Dec 2023 10:00:48 GMT

Dream of Dutch Center for RNA Therapeutics in 'Best Idea' of the Netherlands and Flanders

The dream of the DCRT has been published on the website of Best Idea in the Netherlands and Flanders! Annemieke Aartsma-Rus, Willeke van Roon-Mom and Marlen Lauffer's contribution is in Dutch and describes DCRT's dream to build a center where patients with a rare genetic disease can be treated with DCRT's personalized therapy to better their quality of life.

posted on December 18, 2023

PhD student Edwin van Oosten presented his poster at the retina degeneration meeting in Torremolinos

Mon, 20 Nov 2023 15:49:50 GMT

Poster presentation by PhD student Edwin van Oosten at the XXth International Symposium on Retina Degeneration in Torremolinos, Spain

At the XXth International Symposium on Retina Degeneration in Torremolinos from 23 - 27 October 2023, PhD student Edwin van Oosten (Radboud University Medical Center) presented his poster entitled 'Development of a customized antisense oligonucleotide-based therapeutic strategy for Stargardt disease'.

posted on November 20, 2023

DCRT presented at the 19th annual meeting of the Oligonucleotide Therapeutics Society in Barcelona

Wed, 25 Oct 2023 11:03:10 GMT

DCRT presented at the 19th annual meeting of the OTS in Barcelona

The DCRT was well represented at the 19th annual meeting of the Oligonucleotide Therapeutics Society in Barcelona. Alex Garanto was co-chair of the organizing committee and further co-chaired the short talk session and the late breaking talks. Rob Collin was a member of the organizing committee and co-chaired the pre-clinical (early phase) session. Anouk Spruit, Marlen Lauffer, Bianca Zardetto and Emilio Harris Mostert presented their poster. Bianca and Emilio even received an OTS poster award! And of course Willeke van Roon-Mom and Annemieke Aartsma-Rus were there to cheer them on :-)

posted on October 25, 2023

Annemieke Aartsma-Rus presents on DCRT and N-of-1 at Eurordis open academy alumni meetup

Wed, 27 Sep 2023 08:00:16 GMT

Annemieke Aartsma-Rus presented on N-of-1 at the EURORDIS open academy

Annemieke presented to a group of patient advocates on the DCRT at the Eurordis open academy alumni meetup on September 22.

posted on September 27, 2023

Join the N1C seminar by Marlen Lauffer on 18 September!

Mon, 04 Sep 2023 07:54:37 GMT

Selecting the right candidate: Which disease-causing variants are eligible for N=1 ASOs?

Individualized genetic treatments provide new opportunities for targeted therapies for rare disease patients. Unfortunately, not all individuals affected by a rare disease qualify for tailored N=1 approaches, and selecting those most likely to benefit is challenging. Patient identification can be divided into the evaluation of (i) the disease-causing variant, (ii) the disease, and (iii) the status of the affected individual. DCRT's Marlen Lauffer will tell you about our efforts to identify eligible variants in the N1C seminar series on 18th September 2023.

posted on September 4, 2023

Marlen Lauffer will give a presentation about DCRT's work at the LUMC Top Research Seminar

Wed, 30 Aug 2023 09:26:02 GMT

Marlen Lauffer will give a presentation about DCRT's research on 19th September 2023

Marlen Lauffer will speak at the Top Research Seminar of the Neuroscience Theme about DCRT's research into individualized RNA treatments for rare neurological disorders. This Top Research Seminar will be held at LUMC on the 19th of September 2023 from 17.00 - 18.30.

Team DCRT outing

Mon, 10 Jul 2023 10:48:33 GMT

Team DCRT @LUMC went on a summer outing to lake Valkenburg

On a beautiful summer's day last week, Team DCRT had a fun outing at a nearby lake. We started with a nice lunch and two rounds of getting to know you questions, followed by an outdoor game called Kubb involving wooden sticks and blocks and a king. It was not as easy as it looked but we had fun!

posted on July 10, 2023

Bioinformatician Redmar van den Berg joins the DCRT

Thu, 08 Jun 2023 08:36:06 GMT

Bioinformatician Redmar van den Berg joins the DCRT

We would like to welcome Redmar van den Berg who has started on June 1st on DCRT's computational project!

Here is his biosketch: Redmar van den Berg is a scientific programmer at the DCRT. He is working on a four year project to develop a tool to predict the effect of mutations on a given protein, and propose possible therapies. Redmar obtained a Master degree in Molecular and Cellular Life Sciences at Utrecht University, with a specialization in bioinformatics. The next 4 years, he worked at the Dutch food safety authority (NVWA), implementing sequencing based analysis of food borne pathogens. This included both outbreak tracing and molecular typing of pathogens, for example by identifying antibiotic resistance genes. He joined the LUMC four years ago, supporting the clinical genetics department, and working on various research projects. He currently splits his time between the DCRT and the Hematology department, where he works on acute leukemia.
https://github.com/Redmar-van-den-Berg

posted on June 8, 2023

Awards for two DCRT PhD students !

Wed, 07 Jun 2023 11:27:52 GMT

Awards for two DCRT PhD students !

At the end of a very successful edition of the Dutch Antisense Therapeutics Symposium, host and co-organizer Alex Garanto presented the DATS award for best poster and best selected talk to young researchers Bianca Zardetto (LUMC) and Emilio Harris-Mostert (EMC). Congratulations! They were also very happy to receive the much desired DATS special edition coffee mug ��.

Timothy Yu from Boston Children's Hospital at the DATS

Wed, 07 Jun 2023 09:47:05 GMT

Dr. Timothy Yu from Boston Children's Hospital at the DATS

The DCRT had invited Dr. Timothy Yu from Boston Children’s hospital to give a keynote lecture at the DATS just before the DCRT session on day 2 of the symposium. Tim is the clinician who developed the first N=1 exon skipping antisense oligonucleotide for a patient with Batten disease with a cryptic splicing mutation (milasen). To make it more attractive for him to come all the way to the Netherlands we promised him stroopwafels and a boat tour in Amsterdam. He agreed and arrived in Leiden the day before the start of the DATS and gave a presentation at the LUMC.

In the evening, we went to Amsterdam for a 3-hour boat tour. Dinner was prepared by restaurants along the route and was served on the boat. Very good food by the way, highly recommended if you are looking for an original evening out.

On Thursday morning we all left by train to Nijmegen for the DATS. Marlen Lauffer, Rob Collin (Radboudumc) and Ype Elgersma (ErasmusMC) gave a talk in the DCRT session. Furthermore, we had organized a panel discussion about N=1 regulatory issues with Valentina Cordo, a representative from the European Medicines Agency. DCRT posters were presented by Laurie Kerkhof, Frederique Rotteveel, Bianca Zardetto, Dion den Hertog and Emilio Harris-Mostert (EMC).

posted on June 7, 2023

DCRT poster presentations at the DATS

Mon, 05 Jun 2023 14:12:12 GMT

Poster presentations by Laurie, Bianca, Dion, Frederique and Emilio

DCRT's (PhD) students Dion den Hertog, Bianca Zardetto, Laurie Kerkhof, Frederique Rotteveel and Emilio Harris-Mostert presented their poster at the 2nd Dutch Antisense Therapeutics Symposium in Nijmegen. They did a great job explaining their research to the attendees. Bianca Zardetto was also selected to give a poster pitch and competed for the first prize in the poster category!

posted on June 5, 2023

Large delegation of the DCRT at the DATS

Mon, 05 Jun 2023 12:49:20 GMT

Large delegation of the DCRT presented at the DATS

The DCRT participated in a special session at the 2nd Dutch Antisense Therapeutics Symposium that was held in Nijmegen, The Netherlands, on 1 and 2 June 2023. The DCRT session, chaired by Willeke van Roon-Mom, consisted of presentations by the three partner institutes LUMC, ErasmusMC and Radboudumc. Marlen Lauffer, Ype Elgersma and Rob Collin presented their work on antisense oligonucleotide therapy. The session concluded with a panel discussion chaired by Annemieke Aartsma-Rus on regulatory issues with Valentina Cordo from the European Medicines Agency. DCRT's early career researchers Laurie Kerkhof, Frederique Rotteveel, Bianca Zardetto and Dion den Hertog presented their poster at the DATS. More on this in further news feeds!

posted on June 5, 2023

Annemieke Aartsma-Rus presented about the DCRT at the 7th Gratama workshop in Nagasaki, Japan

Wed, 24 May 2023 11:17:07 GMT

Annemieke Aartsma-Rus presented the DCRT at the 7th Gratama workshop in Japan

At the 7th Gratama workshop that was held from 10-12 May 2023 in Nagasaki, Japan, Annemieke Aartsma-Rus presented the activities of the DCRT consortium.

Marlen Lauffer represented DCRT at Biotech Wednesday

Thu, 11 May 2023 11:36:56 GMT

Marlen Lauffer represented DCRT at the HollandBIO event in Utrecht

On May 10th, 2023, Marlen Lauffer gave a presentation at the HollandBIO event 'Biotech Wednesday: Going for the Cure'. She presented DCRT's work on individualized RNA treatments for patients with nano-rare neurological disorders. Thanks to HollandBIO for the nice photo!

posted on May 11th, 2023

New student Frederique joins DCRT

Mon, 13 Mar 2023 13:05:36 GMT

New student Frederique joins DCRT

My name is Frederique Rotteveel and during my internship I will be helping Laurie Kerkhof with the screening and analysis of antisense oligonucleotides. The aim is to develop possible therapies for spinocerebellar ataxia 1 (SCA1).

I obtained my bachelors degree in Biomedical Sciences at the University of Amsterdam where I followed the Neurobiology minor and multiple Psychology courses. Currently I am living in Leiden where I just started my master’s degree. I am excited to be joining the DCRT team for the upcoming 5 months!

posted on March 13, 2023

DCRT celebrates third anniversary!

Wed, 01 Mar 2023 10:30:06 GMT

DCRT celebrates its third anniversary on Rare Disease Day!

DCRT was founded three years ago on rare disease day (28th February). We have made a lot of progress and are happy to continue our research into individualized ASO therapy for patients with a rare, genetic disease of the brain or eye.

posted on 28th February 2023

DCRT was represented by Annemieke Aartsma-Rus at a meeting of the European Medicines Agency (EMA) on RNA therapy

Thu, 09 Feb 2023 08:54:25 GMT

DCRT was represented by Annemieke Aartsma-Rus at a meeting of the European Medicines Agency (EMA) on RNA therapy

On February 2nd Annemieke Aartsma-Rus presented at a meeting on RNA therapy organized by the European Medicines Agency (EMA). She presented the state of the art for single stranded oligonucleotides, how splice modulating oligonucleotides can be exploited for individual mutations for patients with progressive brain diseases, and introduced how these are being developed within the setting of the DCRT and the 1 Mutation 1 Medicine network.

During the panel discussion and the EMA press conference on February 3 ^rd she stressed the importance of starting a dialogue with regulators early, especially for academics as they have generally very little expertise with the regulatory system. She underlined her positive interaction with the Innovation Task Force and constructive feedback provided to the DCRT.

posted on February 9, 2023

Annemieke Aartsma-Rus participated in a panel discussion on the future of RNA technology at the European Parliament

Wed, 25 Jan 2023 11:57:43 GMT

Annemieke Aartsma-Rus participated in a panel discussion about the future of RNA-based technology at the European Parliament

On 25th January, Annemieke Aartsma-Rus (second from the left in the photo) joined the Panel for the Future of Science and Technology (STOA) for a roundtable discussion on the future of RNA therapy. On this occasion, Annemieke has been interviewed for the European Science-Media Hub. You can read the interview here .

posted on January 25, 2023

DCRT article on the development of tailored splice switching oligonucleotides for progressive brain disorders in Europe

Mon, 23 Jan 2023 13:57:36 GMT

DCRT's latest article on the development of tailored splice switching oligonucleotides for progressive brain disorders in Europe

This article that we wrote with our colleagues from Tuebingen describes the state of the art in ASO treatment options for rare neurological diseases. Both 1 Mutation 1 Medicine (1M1M) and Dutch Center for RNA Therapeutics (DCRT) aim to develop patient-specific ASOs and treat eligible patients within Europe and the Netherlands, respectively. The article offers insight into the challenges and advantages of this type of treatment and describes regulatory issues, patient eligibility criteria, safety studies, ASO development, envisioned treatment and measuring clinical outcomes. It gives a very clear overview of what DCRT and 1M1M have done to prepare for treatment of eligible patients.

posted on January 23, 2023

Master student Dion den Hertog joins DCRT

duda-wsm@mijndomein.nl — Wed, 11 Jan 2023 09:28:02 GMT

Master student Dion den Hertog joins DCRT

Dion den Hertog is a master student Biomedical Science at Leiden University, now doing an internship at the DCRT under the supervision of Bianca Zardetto. He aims to develop a disease relevant platform to reliably test personalized and generalized ASOs. This platform will be created using transdifferentiation of patient derived fibroblasts to oligodendrocytes.

Dion obtained his bachelor’s degree in Biology and Medical Laboratory Research in Rotterdam. He did his bachelor internship at the lab of Prof. Ype Elgersma where he researched the effect of UBE3a on behaviour in Angelman and Duplication 15q mice models.

https://www.linkedin.com/in/dion-den-hertog-121a501a2

posted on January 11, 2023

Read the new DCRT publication!

duda-wsm@mijndomein.nl — Wed, 04 Jan 2023 11:35:40 GMT

Read the new DCRT publication in the OTS edition of Nucleic Acid Therapeutics!

DCRT's Annemieke Aartsma-Rus, Willeke van Roon-Mom and Alex Garanto wrote the Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides together with other members of the N=1 Collaborative.

Merry Christmas and a Happy New Year!

duda-wsm@mijndomein.nl — Thu, 22 Dec 2022 13:35:50 GMT

Merry Christmas and a Happy New Year!

From all of us at DCRT merry Christmas and a happy and succesful New Year!

posted on December 22, 2022

Student Quint Blom joins DCRT

duda-wsm@mijndomein.nl — Thu, 08 Dec 2022 13:24:20 GMT

DCRT welcomes student Quint Blom

I am Quint Blom. I am born in Hilversum, but currently living in the beautiful Leiden. I am a ‘Biology & Medical Laboratory Work’ student at the Applied Sciences Leiden. I just finished my minor ‘Biomedicine/Biotechnology’ at the Francisco de Victoria Universidad in Madrid, Spain which was a very nice experience. I am excited to be joining the DCRT team for the upcoming 9 months during my internship! I’ll be mainly focusing on the toxicology of different antisense oligonucleotides therapies.

posted on December 8, 2022

DCRT said goodbye to Iris Huitink

duda-wsm@mijndomein.nl — Wed, 30 Nov 2022 10:50:18 GMT

Time to say goodbye to stud ent Iris Huitink who finished her internship at DCRT

Iris Huitink finished her internship with DCRT and is continuing her Master studies in Amsterdam. She has developed the ExonCheck tool, a very important step in the assessment of single variants. To thank her for her hard work, we took her out for lunch. And of course we wish her all the best in her future career!

posted on November 30, 2022

Annemieke Aartsma-Rus represented DCRT at the meeting of the N=1 Collaborative

duda-wsm@mijndomein.nl — Tue, 18 Oct 2022 12:54:33 GMT

Annemieke Aartsma-Rus represented DCRT at the first in-person meeting of the N=1 Collaborative

Annemieke Aartsma-Rus represented the DCRT at the first in-person meeting of the N=1 Collaborative in Phoenix on October 6, 2022. The meeting was attended by core members of the collaborative such as Tim Yu, Jon Watts, Julia Vitarello (Mila's mother) and Keith Flanagan.

posted on October 18, 2022

Annemieke Aartsma-Rus presented on the DCRT at the Agora meeting in London on September 22nd

duda-wsm@mijndomein.nl — Thu, 06 Oct 2022 09:56:16 GMT

Annemieke Aartsma-Rus presented on the DCRT at the AGORA meeting in London on 22nd of September

Annemieke was invited to give a presentation on DCRT's oligonucleotide therapy at the first meeting of the AGORA consortium in London on 22 September. AGORA focuses on access to treatment for children with ultra-rare diseases which mainly include inborn errors of immunity or metabolism.

posted on October 6, 2022

DCRT's postdoc Marlen Lauffer wins poster prize at the OTS

duda-wsm@mijndomein.nl — Thu, 06 Oct 2022 09:11:42 GMT

DCRT's Marlen Lauffer wins poster prize at the OTS conference

At the OTS conference in Phoenix this week, Marlen Lauffer won a prize for her poster on DCRT's ExonCheck tool that has been developed by Marlen's student Iris Huitink to screen single variants for possible ASO-based exon skipping. Well done, Marlen, excellent content, design and colors!

posted on October 6, 2022

Student Sowmiyaa Kumar joins the DCRT

duda-wsm@mijndomein.nl — Wed, 28 Sep 2022 12:04:57 GMT

Welcome to DCRT's new student Sowmiyaa Kumar

I am Sowmiyaa Senthil Kumar, and I am a student at the Vrije Universiteit Amsterdam in the Master's program of Bioinformatics and Systems Biology. I will be joining the DCRT for a 6 months' traineeship to continue and expand the work of Iris Huitink on the ExonCheck tool. ExonCheck provides information linked to a rare pathogenic variant which allows the user to make an informed decision of whether the said variant is suitable for RNA ASO therapy.

posted on September 28, 2022

Annemieke Aartsma-Rus gave a talk about DCRT's RNA therapy

duda-wsm@mijndomein.nl — Wed, 07 Sep 2022 10:00:51 GMT

Annemieke Aartsma-Rus gave a talk in Malaga about DCRT's RNA therapy

Annemieke Aartsma-Rus gave a presentation entitled 'RNA based therapies for rare disease treatment' at the 44th SEBBM congress in Malaga this week. Here is a photo of Annemieke with Madrid colleague Lourdes Desviat (UAM).

DCRT’s postdoc Marlen Lauffer gave a course on neurodevelopment and neurodegeneration at a summer school in Vienna

duda-wsm@mijndomein.nl — Mon, 29 Aug 2022 09:20:40 GMT

DCRT's postdoc Marlen Lauffer gave a course on neurodevelopment and neurodegeneration at a summer school in Vienna

Summer School ‚The beginning and end of a neuron’

From 25 ^th July to 3 ^rd August 2022, DCRT’s postdoc Marlen Lauffer, together with her friend and colleague Eliana Nachman (Postdoc, VIB Center for Brain & Disease Research - KU Leuven), was teaching a course on neurodevelopment and neurodegeneration at a summer school in Vienna.

The course, aimed at undergraduate students from the life sciences and medicine, covered a broad range of topics from neurodevelopment and neurodevelopmental disorders to neurodegeneration and neurodegenerative diseases. The students further learnt about modelling systems for neurological disorders and therapeutic options, the ones already on the market and new developments. Multiple interactive sessions gave the students the possibility to act as representatives of a pharma company, an ethic’s committee, a patient organization or a regulatory agency, and the option to design a pre-clinical and clinical testing pipeline for imaginary neurological diseases. One session was also dedicated to the DCRT and the possibilities and difficulties of n-of-1 treatments.

As part of the summer school, Marlen and Eliana got the chance to talk in front of all 140 participants giving a talk titled “A therapy for every(one) in a million?”. Here, they scripted a stage fight contrasting the need and costs for therapies targeted at a single person (one in a million) with costs and need for therapies targeting neurodegenerative diseases like Alzheimer’s (everyone in a million).

The summer school was a full success, and the students could not have formulated it better in their final session “Despite having lots of fun, we learnt a lot!”

posted on August 29, 2022

DCRT team in front of the LUMC research building

duda-wsm@mijndomein.nl — Wed, 24 Aug 2022 11:04:30 GMT

DCRT team in front of the LUMC research building

Greetings on this beautiful summer day from the Dutch Center for RNA Therapeutics at Leiden University Medical Center. From left to right standing: Willeke van Roon-Mom, Annemieke Aartsma-Rus, Pauline de Graaf, Marlen Lauffer. From left to right sitting: Bianca Zardetto, Anouk Spruit, Laurie Kerkhof. Together with our partner institutes in Rotterdam and Nijmegen we aim to develop RNA therapy for ultra-rare genetic disorders of the brain or the eye.

posted on August 24, 2022

DCRT presentation at the FEBS session on RNA therapeutics for genetic and infectious disease

Thu, 21 Jul 2022 09:58:09 GMT

DCRT presentation at the FEBS session on RNA therapeutics for genetic and infectious disease session

Annemieke Aartsma-Rus presented about the DCRT during the RNA therapeutics for genetic and infectious disease seminar that was organized during the BioSummit by FEBS, IUBMB and PABMB in Lisbon Portugal on July 12 ^th . The session was chaired by Prof Fragkoulis and Prof Viguera and featured an introduction to RNA therapies by Prof Desviat (via zoom due to COVID), an overview of opportunities for DNA and RNA vaccines by Prof Pavlakis and the concept of developing individualized antisense oligonucleotides by Prof Aartsma-Rus. The session was very well attended and was followed by a lively discussion with the audience.

posted on July 21, 2022

Professor Willeke van Roon-Mom received the 3rd Riccardo Cortese's Lecture award at the SIBBM in Rome

duda-wsm@mijndomein.nl — Wed, 20 Jul 2022 07:55:00 GMT

Professor Willeke van Roon-Mom received the 3rd Riccardo Cortese's Lecture award at the SIBBM in Rome

At the SIBBM 2022 conference 'Frontiers in molecular biology, the RNA world 3.0', Willeke van Roon-Mom received an award for her lecture on the DCRT. A big thank you to the organization. We are very honoured!

posted on July 20, 2022

Professor Rob Collin's inaugural lecture at Nijmegen University

Thu, 14 Jul 2022 12:09:21 GMT

Professor Rob Collin gave his inaugural lecture on the 8th of July 2022 at Radboud University in Nijmegen

Professor Rob Collin is preparing/dressing for his inaugural lecture at Radboud University in Nijmegen. It is official now! Congratulations from your DCRT colleagues!

posted on July 14, 2022

Best presentation abstract at NVGCT for Dyah Karjosukarso

duda-wsm@mijndomein.nl — Thu, 14 Jul 2022 11:15:13 GMT

Best Abstract award from the ESGCT for Dyah Karjosukarso

At the annual symposium of the NVGCT in June 2022, Dyah received another award: the ESGCT Best Abstract award for her presentation entitled "Towards clinical application of antisense oligonucleotides for the treatment of Stargardt disease".

posted on July 14, 2022

Dyah Karjosukarso and Irene Vázquez-Domínguez were awarded best poster and best presentation at the DATS

duda-wsm@mijndomein.nl — Thu, 14 Jul 2022 10:57:36 GMT

DCRT's Dyah Karjosukarso and Irene Vázquez-Domínguez received an award for best poster and best short talk at DATS

DCRT's Dyah Karjosukarso and Irene Vázquez-Domínguez (Radboudumc) were presented with an award for best poster and best short talk at the 1st Dutch Antisense Therapy Symposium on June 3, 2022. Congratulations!

posted on July 14, 2022

OTS young investigator award 2021 for Alex Garanto

duda-wsm@mijndomein.nl — Thu, 14 Jul 2022 10:31:13 GMT

OTS Young Investigator Award 2021 for Dr. Alex Garanto

At the (virtual) OTS conference last September, Dr. Alex Garanto received the Young Investigator Award for his outstanding achievements and contributions in the field of oligonucleotide therapeutics. Congratulations!

posted on July 14, 2022

DCRT's PhD student Laurie Kerkhof at the 1st Dutch Antisense Therapeutics Symposium

duda-wsm@mijndomein.nl — Mon, 20 Jun 2022 09:48:26 GMT

DCRT's PhD student Laurie Kerkhof at the 1st Dutch Antisense Therapeutics Symposium

1 ^st Dutch Antisense Therapeutics Symposium by Laurie Kerkhof

On the 3 ^rd of June, the DCRT was present at the 1 ^st Dutch Antisense Therapeutics symposium in Leiden. The symposium started with an interesting talk from Annemieke Aartsma-Rus (LUMC) on the development of antisense oligonucleotide therapies, elaborating on some of the antisense oligonucleotide modifications that improve drug stability and efficacy. The rest of the morning session consisted of several interesting talks on peptide-based delivery systems and exon-skipping therapies for dystrophic epidermolysis bullosa and CADASIL. The morning session ended with a talk from Marlen Lauffer (LUMC) on N=1 personalized treatments and how the DCRT contributes to this goal.

The afternoon session started with presentations from invited speakers and early career investigators, including Irene Vazques-Dominguez (Radboudumc) and Claudia Milazzo (Erasmus MC), and a nice talk from the international keynote speaker Bruno Godinho (Atalanta Therapeutics) on the delivery of stabilized siRNA scaffolds to the central nervous system. The session continued with talks from a different perspective: Marjon Pasmooij (Medicines Evaluation Board) on regulation of antisense medicine and Elizabeth Vroom (Duchenne Parent Project Netherlands) on the role of patient organizations in drug development.

The talks were followed by poster presentations from Master and PhD students. The poster session was really interactive and a lot of people came to see the posters. This led to lively discussions and also to new insights and ideas.

The day ended with the awards for the best short talk and the best poster, which went to Irene Vasquez-Dominguez (Radboudumc) and Dyah Karjosukarso (Radboudumc), respectively, who both did a really great job! After an interesting day of science, the symposium concluded with networking drinks and a nice dinner. All in all, we look back on a really great and informative symposium. It was a perfect day to network and meet a lot of amazing scientists who are very willing to help out wherever they can!

posted on Jun 20, 2022

Keynote speaker Annemieke with Princess Beatrix at the Muscles2meet symposium

duda-wsm@mijndomein.nl — Mon, 20 Jun 2022 08:12:28 GMT

Annemieke with Princess Beatrix at the Muscles2Meet symposium

Muscles2Meet is a biennial symposium for all PhD students in the Netherlands working on neuromuscular diseases sponsored by the Princes Beatrix Spierfonds. Also Patroness Princess Beatrix attended and Annemieke Aartsma-Rus, keynote speaker at the symposium, had the honour of meeting the former queen of the Netherlands.

posted on Jun 20, 2022

DCRT at the 1st Dutch Antisense Therapy Symposium

duda-wsm@mijndomein.nl — Thu, 09 Jun 2022 09:49:32 GMT

DCRT at the 1st Dutch Antisense Therapeutics Symposium

The DCRT was very well represented at the 1st Dutch Antisense Therapeutics symposium in Leiden on June 3rd. Speakers included Marlen Lauffer (LUMC), Irene Vazquez-Dominguez (Radboudumc) and Claudia Milazzo (ErasmusMC). DCRT's PhD students presented 10 posters in total!

posted on June 9, 2022

Annemieke Aartsma-Rus presented on the DCRT at the Japanese Society of Neurology meeting

duda-wsm@mijndomein.nl — Wed, 25 May 2022 12:27:07 GMT

Annemieke Aartsma-Rus presented on the DCRT at the Japanese Society of Neurology meeting on May 19th

Annemieke also co-chaired a satellited symposium on N-of-1 treatment with antisense oligonucleotides with Takenori Yokota from Tokyo University. Annemieke presented on the Dutch approach to N-of-1 ASO treatment as well as European and global efforts to align N-of-1 ASO development. During the panel session considerations for patient selection and different regulatory aspects in different jurisdictions were discussed.

posted on May 25, 2022

Student Iris Huitink joins DCRT

duda-wsm@mijndomein.nl — Mon, 23 May 2022 10:49:38 GMT

Student Iris joins DCRT

My name is Iris Huitink and I am a master student Bioinformatics and Systems Biology at the Vrije Universiteit Amsterdam. Recently, I started my research project at the DCRT. The DCRT focuses on personalized RNA therapies for patients with very rare diseases. However, the assessment whether a patient is suitable for personalized RNA therapy is still done manually. Therefore, I will work on building a new tool called ExonCheck that facilitates assessing whether a patient’s rare variant is suitable for RNA therapy.

posted on May 23, 2022

Laurie and Bianca: PhD students

duda-wsm@mijndomein.nl — Wed, 18 May 2022 12:37:59 GMT

Two new PhD students, Laurie en Bianca, joined the DCRT and will focus on the development of RNA therapeutics for rare diseases

We are Laurie Kerkhof and Bianca Zardetto, two new PhD students at the DCRT as part of the PSIDER consortium. The PSIDER consortium focuses on the development of human iPSC neuronal platforms that can be used for therapeutic discovery of neurodevelopmental disorders. In our projects, we will work on rare neurodevelopmental and neurodegenerative disorders and will look for mutations that are potentially amenable for correction by antisense oligonucleotide (ASO) therapy. To facilitate the therapeutic discovery of these rare diseases, stem cells will be generated from patient’s cells and differentiated into brain cells. These brain cells will then be used in a 2D and 3D platform on which the mutation specific ASO can be tested. With the help of these patient-derived cellular disease platforms, we will subsequently investigate whether the function of the brain cells can be restored after ASO delivery.

posted on May 18, 2022

Annemieke Aartsma-Rus presented at the DIA/FDA meeting

duda-wsm@mijndomein.nl — Wed, 18 May 2022 10:28:17 GMT

Annemieke Aartsma-Rus presented at the DIA-FDA meeting on oligonucleotides

Annemieke Aartsma-Rus presented on the Dutch Center for RNA Therapeutics at the DIA FDA meeting on oligonucleotides during the session on Individualized Antisense Oligonucleotide Products on April 26. She explained the progress made in the past year and also the collaborations with the 1 mutation 1 medicine consortium and the N-of-1 collaborative .

posted on May 18, 2022

Gene list of interest

Wed, 30 Mar 2022 12:45:02 GMT

The gene list - eligible genes and diseases

The DCRT has compiled a list of genes and diseases that we consider eligible for RNA therapy. This is by no means a comprehensive list, but a starting point for clinicians and researchers to get an idea what conditions we can target with personalized antisense oligonucleotides.

You can find the list following the link below:
DCRT gene list

posted on Mar 30, 2022

Student Max joins the DCRT

Wed, 23 Mar 2022 12:48:05 GMT

Student Max joins the DCRT and focusses on allele-specific anti-sense oligonucleotide treatment

My name is Max Voslamber, a 3rd-year bachelor’s student in biomedical sciences at Leiden University. For the coming 4 months, I will be following an internship at the DCRT where I will be working on the development of an allele-specific antisense oligonucleotide (AON). This project aims to target and inactivate a disease-causing mutated allele but leave the wild-type homologous allele intact. Eventually, the goal is to decrease the disease burden associated with this mutated allele by preventing the translation of the mutated transcript using an AON. During my stay at the DCRT, I will be most focused on performing trans-differentiation, midi-gene assays and RNA quantification, which will be used to test and evaluate the AONs. I will tell you more about the assays later on.

published on Mar 23, 2022

Nieuwe alinea

DCRT anniversary!

Mon, 28 Feb 2022 16:45:03 GMT

Happy anniversary! DCRT turned 2 today! ��

Time flies! The DCRT was officially founded 2 years ago by Professor Annemieke Aartsma-Rus (LUMC), Professor Willeke van Roon-Mom (LUMC), and Professor Rob Collins (RUMC), later joined by Professor Ype Elgersma (EMC). Inspired by the N=1 anti-sense oligonucleotide therapy Milasen against Batten's disease, they set out to start a center of their own, providing customized RNA therapies to patients in the Netherlands. Their extensive backgrounds and expertise in antisense oligonucleotide therapeutics are the basis for developing therapies for patients with an ultra-rare disease. In the past 2 years, the DCRT family has steadily grown, with many people dedicated to delivering bespoke drugs to patients. The DCRT strives for an antisense oligonucleotide therapy for Dutch patients but aims to share all the knowledge gained along the way. Did you know there is also an international N=1 Collaborative (n1collaborative.org) of clinician-scientists, researchers, patients, and companies focused on individualized medicine?

published on Feb 28, 2022

Rare disease day

Wed, 23 Feb 2022 15:45:39 GMT

DCRT supports rare disease day

Members of the DCRT came together to show their support for those 300 million people over the world living with rare diseases and their families. The Dutch Centre of RNA therapy focusses on these patients and specifically on those with ultra rare diseases. We believe anti-sense oligonucleotide therapy could help some of these patients. The photo already gives away that many people are involved in the DCRT consortium. Thank you all for your involvement and support!

published on Feb 23, 2022

Listen now to the gene Fixers podcast!

Fri, 04 Feb 2022 11:25:20 GMT

Gene fixers podcast - anti-sense oligonucleotide therapy

Would you like a better understanding of anti-sense oligonucleotides and gain a better understanding of the difference between Anti-sense oligo nucleotides? Tim Yu who designed the personal therapy Milasen and Annemieke Aarstma-Rus who is in the oligonucleotide field for more than 20 years have an open discussion about anti-sense oligo nucleotide personalized treatments.

You can listen to the postcast via this link .

published on Feb 4, 2022

Annemieke Aartsma-Rus chair for 2022-2024

Thu, 03 Feb 2022 15:51:04 GMT

Annemieke Aartsma-Rus is the elected DCRT chair 2022-2024

Prof. dr. Annemieke Aartsma-Rus was elected as the new chair of the DCRT for the year 2022-2024. Prof. dr. Rob Collin is chosen as the Vice-Chair. Both are very excited to see what the DCRT will accomplish in the coming two years! Annemieke is also involved in the international rare disease research consortium and highlights what they have accomplished in the last 10 years in her #apaperaday on twitter.

published on Feb 3, 2022

One-day symposium of the Centre DNA Fingerprinting and Diagnostics (CDFD), Hyderabad

Thu, 27 Jan 2022 14:16:28 GMT

DCRT presents ''oversees'' in Hyderabad and London

The Centre for DNA Fingerprinting and Diagnostics (CDFD), Hyderabad (India), organized a one-day symposium on the 27th January 2022 with some great speakers. Prof. Dr. Annemieke Aartsma-Rus was invited and presented the work and future plans of the DCRT. Would you like to listen to her talk? You can find the link to the recording in the Events & Talk section.

It has been a busy week as Annemieke also presented at the Festival of Genomics and Biodata (London, UK) on Thursday 26th January. This highlights one of the DCRT’s goals, namely to spread knowledge and educate researchers and clinicians worldwide on the work we do. For more talks on the DCRT, please check our website regularly!

published on Jan 27, 2022

Happy new year!

Fri, 07 Jan 2022 17:28:52 GMT

Happy new year!

The DCRT group wishes you a healthy and happy 2022!

Let it be a great scientific year!

Annemieke received the NVGCT award 2021!

Wed, 10 Nov 2021 12:54:02 GMT

Annemieke Aartsma-Rus received the outstanding achievement award from the Dutch society of gene and cell therapy

The NVGCT is dedicated to promoting basic and clinical research in the field of gene therapy, cell therapy and genetic vaccines in The Netherlands since 1999.

On November 9 2021 Annemieke Aartsma-Rus, co-coordinator of the DCRT, received the outstanding achievement award from the Dutch Society of Gene and Cell Therapy from Rob Collin, board member of the Society and co-coordinator of the DCRT. Annemieke received this award for her pioneering work in developing exon skipping for Duchenne muscular dystrophy, helping others to initiate and further exon skipping for other genetic diseases and for her active role in networking with collaborators and patients.

Congratulations Annemieke!

published on Nov 10, 2021

DCRT at workshop of the DARTER COST Action

Thu, 29 Jul 2021 08:34:07 GMT

DCRT at workshop of the DARTER COST Action

No less than three DCRT coordinators actively contributed to the workshop of the Delivery of Antisense RNA Therapy (DARTER) COST Action that took place from July 19-21. DARTER is a network that aims to foster and facilitate antisense oligonucleotide research in Europe by achieving consensus on which model systems to use to improve delivery and study safety and toxicity of oligonucleotide therapies. COST Actions are funded by the cooperation of science and technology (COST. These networks are open to new participants also after the network has initiated. For more information on DARTER look on the antisenserna website .

During the workshop Willeke van Roon-Mom presented on delivery to brain and cell and animal model systems to test this. Rob Collin presented on delivery to the eye and the development of antisense oligonucleotide therapies for genetic retinal diseases. Finally, Annemieke Aartsma-Rus presented in the “negative results” session. DARTER and DCRT strongly believe that it is important to publish also about hypotheses and ideas that turned out not to work. Sharing these findings will avoid unnecessary duplication of work. Furthermore, hypotheses that are disproven also advance science.

published on Jul 29, 2021

Upcoming Oligonucleotide Therapeutics Society (OTS) meeting in september

Fri, 16 Jul 2021 06:13:39 GMT

Upcoming OTS meeting

Several members of the Dutch Center of RNA Therapeutics will attend the annual meeting of the Oligonucleotide Therapeutics Society (OTS, September 26-29, virtual). The OTS is a non-profit society aiming to advance the development of oligonucleotide therapies, such as the splice modulation approaches the DCRT is pursuing.

The OTS has members from academia and industry and has a strong focus on science and the state of the art of the field is presented each year.

This year there will be a session focusing on developing therapies for very small patient groups. Furthermore, DCRT member Annemieke Aartsma-Rus is part of the organizing committee of the meeting as well as president of the OTS.

We hope to see you virtually at the OTS 2021 Annual meeting .

posted on Jul 16, 2021

PSIDER grant application approved!

Wed, 07 Jul 2021 13:11:36 GMT

PSIDER grant application approved

Recently, the ZonMw-PSIDER grants were awarded that are intended to stimulate stem cell research for rare disorders. DCRT members Ype Elgersma, Annemieke Aartsma-Rus, and Willeke van Roon-Mom are joining forces on the project 'Towards a human iPSC neuronal platform for neurodevelopmental disorder therapeutic discovery.', which is a collaboration between Erasmus MC, Leiden UMC, Utrecht UMC and Nijmegen UMC. The members received 4 million euro.

In this project, patients with a developmental disorder are identified who carry a mutation that is potentially amenable for correction by antisense oligonucleotide (ASO) therapy. In order to develop and test such a personalized medicine, stem cells are generated from the patient's blood cells. These stem cells are then differentiated into brain cells, on which the mutation-specific ASO will be tested to investigate whether the function of the brain cells can be restored.

The researchers hope that this study is the prelude to the routine development of tailor-made treatment for a large group of serious conditions that are hitherto untreatable. In the development of such therapies, DCRT is a very important partner.

published on Jul 7, 2021

Academic Pharma is key to support an effective drug development system for rare diseases

Wed, 07 Jul 2021 07:54:01 GMT

Press release on LUMC.nl

Annemieke Aartsma-Rus featured in a press release on the LUMC website last week.

In this she speaks about the role of Academic Pharma in supporting the process of delivering drugs that are not interesting for pharmaceutical companies, because of the low numbers of patients. Also, the role of DCRT in this process is raised.

To read the complete press release, go to Academic Pharma is key to support an effective drug development system for rare diseases on the LUMC website.

published on Jul 7, 2021

Developing genetic treatments for patients with very rare mutations

duda-wsm@mijndomein.nl — Tue, 25 May 2021 06:27:38 GMT

Developing genetic treatments for patients with very rare mutations

Annemieke Aartsma-Rus published an article about DCRT in Nature Medicine! In this article Annemieke tells about "N of 1" therapies, RNA therapies that are only suitable for one patient. DCRT is developing individualized ASOs for

patients with genetic brain or eye disorders and Annemieke summarizes the challenges that we face in these studies.

To read the whole article, go to Nature Medicine Worldview of 24th of May 2021 .

published on May 25, 2021

Blog: Rare disease

Fri, 30 Apr 2021 12:51:46 GMT

Blog: Rare disease

There are diseases so rare that hardly anyone has ever heard of them. That can change. For example, this time last year, none of us had heard of COVID-19, while everyone is now familiar with it. This is different for hereditary rare diseases. They remain rare. This makes it difficult to study them or develop a therapy for them.

Hereditary diseases often arise due to the lack of a protein. Due to an error, a gene encoding a protein becomes unreadable. The exon-skip approach, a technique I have been working on for 20 years, tries to make mutated genes readable again with a chemically modified piece of DNA, an ASO. This allows patients to make the missing protein again and disease progression can be inhibited.

Friendly environment

ASOs injected into the spinal fluid through an epidural are taken up by the nerve cells of the spinal cord and brain. The spinal cord and brain are a very "friendly" environment for ASOs. Here they are broken down much less quickly than in the rest of the body, so that they have a longer effect. This makes it possible to give the epidural to the patients once every 3-4 months. We know that ASOs administered in this way can be therapeutic from Nusinersen, an ASO drug used to treat patients with spinal muscle atrophy (SMA) worldwide.

Unique therapy

SMA is a rare disease, but it is a relatively common rare disease. There are hundreds of SMA patients in the Netherlands. There are even much rarer diseases. Some of these patients have gene errors that would be well suited for the exon skip approach. However, these are often unique cases and pharmaceutical companies are currently not interested in developing drugs for single patients . Tim Yu, a neurologist from Boston, has shown it is possible to develop an ASO therapy for these patients. For a patient with Batten, a rare metabolic disease, he developed an exon-skip ASO unique to its mutation. This made the mutated gene readable again and allowed her to make the missing protein in the brain.

One year after diagnosis, he had tested the ASO in the patient's cultured cells, in rats (to test for safety), and injected the ASO into the patient's spinal fluid for the first time. It seemed to have an effect. Before treatment, the patient had many seizures on a daily basis, since treatment seizures have decreased in both frequency and duration.

Join efforts

When hearing this story, researchers from several Dutch academic centers with ASO experience thought: we can do that too! The forces have now been joined in the Dutch Center for RNA Therapeutics (DCRT) which includes the LUMC, the Radboudumc and Erasmus MC. The aim of DCRT is to develop ASOs for patients with unique gene errors, where ASO treatment should theoretically work and local treatment is possible. DCRT would offer this treatment at cost price.

Update

We started on Rare Disease Day, February 29, 2021. Meanwhile, progress is being made with the design and testing of ASOs in cultured cells and we are setting up the organizational structure of DCRT. We are pioneers in the Netherlands. We are in contact with people like Tim Yu, but it turns out that the laws and regulations in America are very different than in Europe for this kind of therapy development.

However, our motto is to solve problems when we encounter them and not let the unknown hold us back. I want to update you on our progress in a future blog, if COVID-19 has hopefully become a very rare disease again.

Summary latest advances in delivery of RNA therapies

Wed, 21 Apr 2021 10:44:27 GMT

Summary latest advances in delivery of RNA therapies

Researchers from the neurodegeneration group in collaboration with other participants from an EU COST Action summarise the latest advances in delivery of RNA therapies.

Drugs that target RNA were considered a curiosity until recently, used mainly in research in rare diseases and personalised therapies. However, this new research field has registered a boost in the last few years, with more than a dozen drugs approved for clinical use and many other in different stages of development. Although rare diseases still constitute a major target, many of these drugs are being developed for common diseases such as hyperlipidemia or certain cancers.

The main hurdle of this type of drugs is the delivery to the relevant tissue or organ after administration and the fact that, on their own, they cannot be taken up efficiently by the cells to do their function.

In an article published recently in EMBO Molecular Medicine (1), a group of researchers working on different aspects of the development of this new class of drugs, summarise the major challenges in their delivery, revise the different options that have emerged in the last few years and the issues related to safety for their use in patients. The authors anticipate that this type of drugs will shortly be in use for one or very few patients (personalised medicine) or to treat common diseases, resulting in further accelerated development.

The co-authors of the article, 22 in total from 14 different countries, form part of a EU COST (Cooperation of Science and Technology) funded network (www.antisenserna.eu), thanks to which they are working together and exchanging their expertise with the final goal to bring beneficial treatments to patients.

published on Apr 21, 2021

1. Hammond SM, Aartsma‐Rus A, Alves S, Borgos SE, Buijsen RAM, Collin RWJ, et al. Delivery of oligonucleotide‐based therapeutics: challenges and opportunities. EMBO Molecular Medicine. 2021.

https://www.embopress.org/doi/full/10.15252/emmm.202013243

1 year celebration and Rare disease day

Thu, 25 Feb 2021 11:34:26 GMT

1 year celebration and Rare disease day

1 year celebration and Rare disease day

Today (28th of February) it is Rare Disease day and exactly on this day DCRT exists 1 year! The best day to go live with this website. There’s yet a lot more to come on this website, but already you will find a lot of information about DCRT.

The last year we’ve been busy with organizing lots of things. The first screening of patient cells is taking place at this moment and collaborations have been started.

DCRT has a lot more to come, so keep following this website to stay up to date about our work.

published on Feb 25, 2021