Antisense Oligonucleotide (AON)-based Therapy for Leber Congenital Amaurosis Caused by a Frequent Mutation in CEP290

Leber congenital amaurosis (LCA) is the most severe form of inherited retinal degeneration, with an onset in the first year of life. The most frequent mutation that causes LCA, present in at least 10% of individuals with LCA from North-American and Northern-European descent, is an intronic mutation...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Collin, Rob WJ, den Hollander, Anneke I, van der Velde-Visser, Saskia D, Bennicelli, Jeannette, Bennett, Jean, Cremers, Frans PM
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group 2012
Pynciau:
Original Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC3381589/
https://ncbi.nlm.nih.gov/pubmed/23343883
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2012.3
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