基因治疗中的病毒和非病毒载体:当前状态和临床前景

Guannan Geng1, Yixin Xu1, Ziying Hu1

  • 1ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Fudan University, Shanghai, China; Institutes of Biomedical Sciences, Fudan University, Shanghai, China; State Key Laboratory of Medical Neurobiology and MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China; MOE Engineering Research Center of Gene Technology, China.

EBioMedicine
|July 2, 2025
PubMed
概括

基因治疗使用病毒和非病毒载体来治疗疾病. 最近在腺相关病毒 (AAV) 和脂质纳米粒子 (LNP) 输送方面的进展显示出治疗遗传疾病的前景.