Article
Choroideremia Gene Therapy
28 Sept 2021
Abstract excerpt
Introduction Choroideremia is a rare X-linked recessive retinal degeneration of photoreceptors, retinal pigment epithelium (RPE), and choroid that progresses in a centripetal manner.1 Choroideremia results from pathologic genetic variants of the CHM gene on the X chromosome with nearly all being loss-of-function null mutations.1 The CHM gene product, Rab escort protein 1 (REP-1), is expressed in all human cells...
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