通过基因替代疗法恢复听力,用于在人类DFNB111聋的小鼠模型中多站点表达的基因

Luoying Jiang1, Shao Wei Hu1, Zijing Wang2

  • 1ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai 200031, China; NHC Key Laboratory of Hearing Medicine, Shanghai 200031, China; State Key Laboratory of Medical Neurobiology and MOE Frontiers Center for Brain Science, Fudan University, Shanghai 200032, China; Institutes of Biomedical Sciences, Fudan University, Shanghai 200032, China.

PubMed
概括

基因疗法对MPZL2基因引起的遗传性听力损失有前途. 在小鼠模型中,AAV-ie-Mpzl2治疗改善了听力,并恢复了耳细胞结构.