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Gene therapy for choroideremia: progress, potential and pitfalls

2 Feb 2025

Abstract excerpt

INTRODUCTION: Choroideremia is a rare disease with a significant disease burden. Gene-supplementation methods for choroideremia gene therapy have been the most successful form of gene therapy thus far. AREAS COVERED: The aim of the current review is to provide an overview of current progress of gene therapy trials to date, with a focus on potential and pitfalls of such trials. We propose a novel end point that...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 39893699
DOI: 10.1080/14712598.2025.2459850

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