Department of Human Genetics, Section Ophthalmogenetics, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
Department of Ophthalmology, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
EMBO Mol Med. 2024 Jan;16(1):4-7. doi: 10.1038/s44321-023-00001-1. Epub 2023 Dec 14.
In the April issue of this Journal, Boffa and coworkers put forward a new therapeutic approach for Gyrate Atrophy of the Choroid and Retina (GACR; OMIM 258870) (Boffa et al, 2023). The authors propose to apply gene therapy to the liver for GACR, a metabolic disease primarily affecting eyesight due to retinal degeneration. Their vision is enthusiastically supported by a News and Views comment in the same issue (Seker Yilmaz and Gissen, 2023). However, based on disease pathology, patient's needs, ethical considerations, therapeutic developmental time lines, and current state of the art of gene therapy for liver and eye, we have a different view on this issue: We argue below that local treatment of the eye is the preferred option for GACR.
在本期杂志的四月刊中，Boffa 及其同事提出了一种治疗脉络膜和视网膜脑苷脂沉积症（GACR；OMIM 258870）的新疗法（Boffa 等人，2023 年）。作者提议对肝脏进行基因治疗，以治疗主要影响视力的代谢疾病——视网膜变性。同一期的一篇新闻和观点评论（Seker Yilmaz 和 Gissen，2023 年）热情地支持了他们的这一设想。然而，根据疾病病理学、患者需求、伦理考虑、治疗开发时间线以及肝脏和眼睛基因治疗的当前现状，我们对这个问题有不同的看法：我们在下面主张，眼局部治疗是 GACR 的首选方案。
Zotero 插件