Knockout and Replacement Gene Surgery to Treat Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa
Brief intro:
- Author: Xuehan Sun, Chen Liang, Yangcan Chen, Tongtong Cui, Jiabao Han, Moyu Dai, Ying Zhang, Qi Zhou and Wei Li
- Journal: Human Gene Therapy
- Doi: https://www.doi.org/10.1089/hum.2023.201
- Publication Date: 2024 Mar 14
Products/Services used in the paper
Quotation shows PackGene:All AAV viruses used in this study were produced by PackGene Biotechnology Company, with an initial dosage of 1E10 vg/mL and a total volume of 500 lL.
Research Field:Retinal abnormality
AAV Serotype:AAV2,AAV8
Targeted organ:eye
Animal or cell line strain:mice
Abstract
Mutations in the rhodopsin (RHO) gene are the predominant causes of autosomal dominant retinitis pigmentosa (adRP). Given the diverse gain-of-function mutations, therapeutic strategies targeting specific sequences face significant challenges. Here, we provide a universal approach to conquer this problem: we have devised a CRISPR-Cas12i-based, mutation-independent gene knockout and replacement compound therapy carried by a dual AAV2/8 system. In this study, we successfully delayed the progression of retinal degeneration in the classic mouse disease model RhoP23H, and also RhoP347S, a new native mouse mutation model we developed. Our research expands the horizon of potential options for future treatments of RHO-mediated adRP.
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