Functional, sustained recovery of hearing in Otoferlin-deficient mice using DB-OTO, a hair-cell-specific AAV-based gene therapy.
使用 DB-OTO（一种基于 AAV 的毛细胞特异性基因疗法）使 Otoferlin 缺陷小鼠的听力得到功能性、持续性恢复。
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101577
Chung Yoojin, Koehler Seth D, Cancelarich Sarah, Gibson Tyler M, Pregernig Gabriela, Becker Lars, Artinian Quynh-Anh, Goodliffe Joseph W, Pan Ning, Quigley Tera M, Senapati Arun, Slade Peter G, Smith Lillian M, So Kathy S, Zhang Xichun, Corrales C Eduardo, Weber Peter, Macdonald Lynn E, Zambrowicz Brian, Economides Aris N, Kyratsous Christos A, Burns Joseph C, Palermo Adam T, Sabin Leah R, Lee John, Valayannopoulos Vassili, Whitton Jonathon P, Drummond Meghan C
