Gene therapy for choroideremia using an adeno-associated viral vector encoding Rab escort protein 1: the REGENERATE open-label trial

Background Choroideremia is an X-linked inherited retinal degeneration that begins in childhood with nyctalopia and loss of peripheral vision, and gradually progresses to blindness in adulthood. Choroideremia is caused by null mutations in the CHM gene, which encodes Rab escort protein 1. Objective...

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Bibliographic Details

Published in:	Efficacy and Mechanism Evaluation
Main Authors:	Jasmina Cehajic-Kapetanovic, Marco P Bellini, Laura J Taylor, Imran H Yusuf, Taha Soomro, Lyndon da Cruz, Robert E MacLaren
Format:	Article
Language:	English
Published:	NIHR Journals Library 2024-05-01
Subjects:	adeno-associated virus choroideremia gene therapy outcome measure outcome measures retinal disease
Online Access:	https://doi.org/10.3310/WARA5730