Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6

Cone-rod dystrophy 6 (CORD6) is caused by gain-of-function mutations in the GUCY2D gene, which encodes retinal guanylate cyclase-1 (RetGC1). There are currently no treatments available for this autosomal dominant disease, which is characterized by severe, early-onset visual impairment. The purpose o...

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Detalles Bibliográficos
Autores principales: Mellen, Russell W., Calabro, Kaitlyn R., McCullough, K. Tyler, Crosson, Sean M., Cova, Alejandro de la, Fajardo, Diego, Xu, Emily, Boye, Sanford L., Boye, Shannon E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10285452/
https://www.ncbi.nlm.nih.gov/pubmed/37361352
http://dx.doi.org/10.1016/j.omtm.2023.05.020

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