NEJM· Luke J. LaffinCleveland Clinic Coordinating Center for Clinical Research, ClevelandStephen J. NichollsVictorian Heart Institute, Melbourne, VIC, AustraliaRussell S. ScottNew Zealand Clinical Research, Christchurch, New ZealandPeter M. CliftonRoyal Adelaide Hospital, Adelaide, SA, AustraliaRenate KoopsAotearoa Clinical Trials, Auckland, New ZealandAshish SarrajuCleveland Clinic Coordinating Center for Clinical Research, ClevelandShweta SinghCRISPR Therapeutics, BostonQiuqing WangKathy WolskiCleveland Clinic Coordinating Center for Clinical Research, ClevelandHuansheng XuJen NielsenNaimish PatelJason M. DuranCRISPR Therapeutics, BostonSteven E. NissenCleveland Clinic Coordinating Center for Clinical Research, Cleveland·· 2026-08-28评分0
CTX310 靶向 ANGPTL3 的 CRISPR-Cas9 基因编辑持久性
Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310
导读
CTX310 通过 CRISPR-Cas9 基因编辑靶向 ANGPTL3 展现出持久的疗效。在临床试验中，单次给药后患者血液中 ANGPTL3 蛋白水平显著且持久降低，并伴随有益的血脂改善。该研究结果支持了一次性基因编辑疗法治疗脂质代谢疾病的潜力。
来源：NEJM · nejm.org