Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies

Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies

<i>Background and objectives</i>: Choroideremia (CHM) is an X-linked recessive chorioretinal dystrophy caused by mutations involving the <i>CHM</i> gene. Gene therapy has entered late-phase clinical trials, although there have been variable results. This review gives a summar...

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Bibliographic Details
Main Authors: Alessandro Abbouda, Filippo Avogaro, Mariya Moosajee, Enzo Maria Vingolo
Format: Article
Language:English
Published: MDPI AG 2021-01-01
Series:Medicina
Subjects:
choroideremia
gene
therapy
clinical trials
stem cells
ataluren
Online Access:https://www.mdpi.com/1010-660X/57/1/64

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