Gene therapy for choroideremia: progress, potential and pitfalls.

Abstract

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 may be clinically meaningful for obtaining regulatory approval in subsequent clinical trials. Additionally, we offer recommendations for further optimization of surgical techniques. Expert opinion Lessons learnt from this phase 3 clinical trial, encompassing optimal vector design, delivery techniques, patient selection criteria, and longterm safety profiles can be used in the development of treatments for polygenic retinal disorders, which may necessitate a more nuanced approach due to genetic complexity.