Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis

Mutations in GUCY2D are associated with severe early-onset retinal dystrophy, Leber congenital amaurosis type 1 (LCA1), a leading cause of blindness in children. Despite a high degree of visual disturbance stemming from photoreceptor dysfunction, patients with LCA1 largely retain normal photorecepto...

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Detalles Bibliográficos
Autores principales: Boye, Sanford L., O’Riordan, Catherine, Morris, James, Lukason, Michael, Compton, David, Baek, Rena, Elmore, Dana M., Peterson, James.J., Fajardo, Diego, McCullough, K. Tyler, Scaria, Abraham, McVie-Wylie, Alison, Boye, Shannon E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9830033/
https://www.ncbi.nlm.nih.gov/pubmed/36654798
http://dx.doi.org/10.1016/j.omtm.2022.12.007

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