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Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?

INTRODUCTION: Choroideremia is an X-linked inherited retinal degeneration resulting from mutations in the CHM gene, encoding Rab escort protein-1 (REP1), a protein regulating intracellular vesicular transport. Loss-of-function mutations in CHM lead to progressive loss of retinal pigment epithelium (...

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Detalles Bibliográficos
Autores principales:	Han, Ruofan Connie, Fry, Lewis E., Kantor, Ariel, McClements, Michelle E., Xue, Kanmin, MacLaren, Robert E.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7610829/ https://www.ncbi.nlm.nih.gov/pubmed/34040899 http://dx.doi.org/10.1080/21678707.2021.1882300

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