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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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Xehetasun bibliografikoak
Argitaratua izan da:Expert Rev Ophthalmol
Egile Nagusiak: Xue, Kanmin, MacLaren, Robert E.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 2018
Gaiak:
Sarrera elektronikoa: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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