Knockout and Replacement Gene Surgery to Treat Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa
简介:
- 作者: Xuehan Sun, Chen Liang, Yangcan Chen, Tongtong Cui, Jiabao Han, Moyu Dai, Ying Zhang, Qi Zhou and Wei Li
- 杂志: Human Gene Therapy
- Doi: https://www.doi.org/10.1089/hum.2023.201
- 出版日期: 2024 Mar 14
论文中使用的产品/服务
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
摘要
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.
关于派真
作为一家专注于AAV 技术十余年，深耕基因治疗领域的CRO&CDMO，派真生物可提供从载体设计、构建到 AAV、慢病毒和 mRNA 服务的一站式解决方案。凭借深厚的技术实力、卓越的运营管理和高标准的服务交付，我们为全球客户提供一站式CMC解决方案，包括从早期概念验证、成药性评估到IIT、IND及BLA的各个阶段。
凭借我们独立知识产权的π-alphaTM 293 细胞AAV高产技术平台，我们能将AAV产量提高多至10倍，每批次产量可达1×10¹⁷vg，以满足多样化的商业化和临床项目需求。此外，我们定制化的mRNA和脂质纳米颗粒（LNP）产品及服务覆盖药物和疫苗开发的各个阶段，从研发到符合GMP的生产，提供端到端的一站式解决方案。
