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
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