Patients and animal models of CNGβ1-deficient retinitis pigmentosa support gene augmentation approach

Retinitis pigmentosa (RP) is a major cause of blindness that affects 1.5 million people worldwide. Mutations in cyclic nucleotide-gated channel β 1 (CNGB1) cause approximately 4% of autosomal recessive RP. Gene augmentation therapy shows promise for treating inherited retinal degenerations; however,...

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Detalles Bibliográficos
Autores principales: Petersen-Jones, Simon M., Occelli, Laurence M., Winkler, Paige A., Lee, Winston, Sparrow, Janet R., Tsukikawa, Mai, Boye, Sanford L., Chiodo, Vince, Capasso, Jenina E., Becirovic, Elvir, Schön, Christian, Seeliger, Mathias W., Levin, Alex V., Michalakis, Stylianos, Hauswirth, William W., Tsang, Stephen H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2017
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5749539/
https://www.ncbi.nlm.nih.gov/pubmed/29202463
http://dx.doi.org/10.1172/JCI95161

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