2025-03-21报道，来自艾伦研究所和西雅图儿童研究所的研究人员在Dravet综合症小鼠模型中合作开发了一种新的基因替代疗法。这种疗法缓解了症状，并导致了长期康复，没有毒性、副作用和死亡。对于那些正在努力应对Dravet综合征挑战的家庭来说，这是 ...

一项由北京协和医院和锦篮基因联合开展的研究，首次聚焦中国健康人群和罕见病患者的AAV9抗体分布。该研究在2022年11月至2024年6月期间，招募了341名参与者，年龄覆盖0至90岁，包括270名健康个体和71名罕见病患者。

Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle ...

To help improve gene therapy, a multidisciplinary team has generated a comprehensive atlas that researchers can use to select ...

The EMA has called a temporary halt to a phase 2 trial of Sarepta's Roche-partnered Duchenne muscular dystrophy (DMD) gene ...

Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle ...

为解决腺相关病毒（AAV）载体中未甲基化的 CpG 二核苷酸引发免疫反应影响治疗效果的问题，研究人员开展优化富含 CpG 的 AAV8 载体 pVR59 的研究。结果得到 CpG 缺失载体 pNC182，维持疗效且降低免疫反应，为 LPLD 治疗及 AAV 载体设计提供思路。 腺相关病毒（Adeno ...

Variant SAS and the Rare Ocular Diseases Center at the University of Campania Luigi Vanvitelli (UCLV) have received positive feedback from the EMA for VAR-002, a recombinant AAV vector gene therapy ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved ...

Financing led by new investor Bain Capital with participation from new investor Wellington Management and all existing investorsProceeds to ...