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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 (...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2021
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Materias: | |
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 |