ROME — In this Healio Video Perspective from the FLORetina-ICOOR meeting, Bart Leroy, MD, PhD, speaks about RNA interference technology for the treatment of Leber congenital amaurosis type 10.
LCA10 is the worst type of Leber congenital amaurosis, with the lowest level of best corrected visual acuity. Leroy explains the genetic basis of this disease, the rationale for antisense oligonucleotide therapy, and the problems encountered with EMA and FDA when trying to design a phase 3 study. The project was eventually abandoned.
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