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
RARE-NL AON meeting @LUMC March 11
Webinar on the use of platform technologies in the non-clinical and clinical domains
Happy new year from all of us at DCRT
Student Roan Hofstede joins DCRT for an internship
Second DCRT newsletter
PhD student Raul Andres Santamaria presented his research at the OTS in Budapest
DCRT's Rob Collin gave a presentation at the annual meeting of the OTS
DCRT at the annual meeting of the N=1 Collaborative
Student Kamala Vermeer joins the DCRT for an internship
DCRT's Marlen Lauffer will give a workshop at the American Society of Human Genetics Meeting in Boston