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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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| Publicado no: | Hum Mol Genet |
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| Main Authors: | , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Oxford University Press
2015
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| Assuntos: | |
| Acesso em linha: | 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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