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...

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Detalles Bibliográficos
Autores principales: Collin, Rob WJ, den Hollander, Anneke I, van der Velde-Visser, Saskia D, Bennicelli, Jeannette, Bennett, Jean, Cremers, Frans PM
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2012
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3381589/
https://www.ncbi.nlm.nih.gov/pubmed/23343883
http://dx.doi.org/10.1038/mtna.2012.3

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