Expert opinion on biological therapy · 2025
Gene therapy for choroideremia: progress, potential and pitfalls.
СтатьяОбзорПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод пока не подготовлен
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Abdalla Elsayed Maram E A, Cehajic-Kepetanovic Jasmina, MacLaren Robert E
Аннотация
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 long-term 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.
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 long-term 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.