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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Bibliographic Details
Main Authors: Rob WJ Collin (Author), Anneke I den Hollander (Author), Saskia D van der Velde-Visser (Author), Jeannette Bennicelli (Author), Jean Bennett (Author), Frans PM Cremers (Author)
Format: Book
Published: Elsevier, 2012-01-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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