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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Detalhes bibliográficos
Publicado no:J Clin Invest
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2017
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5749539/
https://ncbi.nlm.nih.gov/pubmed/29202463
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI95161
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