Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
<p><strong>Introduction</strong>: Choroideremia is an X-linked inherited retinal degeneration resulting from mutations in the <em>CHM</em> gene, encoding Rab escort protein-1 (REP1), a protein regulating intracellular vesicular transport. Loss-of-funct...
Main Authors: | , , , , , |
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Format: | Journal article |
Language: | English |
Published: |
Taylor and Francis
2021
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