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Ocular gene therapy for choroideremia: clinical trials and future perspectives
INTRODUCTION: Gene therapy offers the potential for targeted replacement of single gene defects in inherited retinal degenerations. AREAS COVERED: Choroideremia is an X-linked blinding retinal disease resulting from deficiency of the CHM gene product, REP1. The disease represents an ideal target for...
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| Publicado no: | Expert Rev Ophthalmol |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6520227/ https://ncbi.nlm.nih.gov/pubmed/31105764 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/17469899.2018.1475232 |
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