A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration

Mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene account for >70% of X-linked retinitis pigmentosa (XLRP) and 15–20% of all inherited retinal degeneration. Gene replacement therapy for RPGR-XLRP was hampered by the relatively slow disease progression in mouse models and by diffi...

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Publicat a:Hum Mol Genet
Autors principals: Wu, Zhijian, Hiriyanna, Suja, Qian, Haohua, Mookherjee, Suddhasil, Campos, Maria M., Gao, Chun, Fariss, Robert, Sieving, Paul A., Li, Tiansen, Colosi, Peter, Swaroop, Anand
Format: Artigo
Idioma:Inglês
Publicat: Oxford University Press 2015
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Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4476444/
https://ncbi.nlm.nih.gov/pubmed/25877300
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv134
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