Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
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DOI: 10.1080/21678707.2021.1882300
Abstract: ABSTRACT 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… read more here.
Keywords: choroideremia; subretinal aav; option choroideremia; gene ... See more keywords