Investigative ophthalmology & visual science۲۰۲۶نمایه شده در PubMed
Catherine E Estay-Ahumada, Michel Roux, Dominique Ciocca, Naima El-Kholti, Marie-Christine Birling, Paola Rossolillo, Marie-Paule Felder-Schmittbuhl, David Hicks
چکیده
Mutations in the gene ABCA4 coding for photoreceptor-specific ATP-binding cassette subfamily A member 4, are responsible for Stargardts disease type 1 (STGD1), the most common form of inherited macular degeneration. We recently showed that injection of viral vectors expressing CRISPR/Cas9 tools…
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DOI: 10.1167/iovs.67.5.47