Single Administration of AAV‐mAtp6v1b2 Gene Therapy Rescues Hearing and Vestibular Disorders Caused by Atp6v1b2‐Induced Lysosomal Dysfunction in Hair Cells

Single Administration of AAV‐mAtp6v1b2 Gene Therapy Rescues Hearing and Vestibular Disorders Caused by Atp6v1b2‐Induced Lysosomal Dysfunction in Hair Cells

Abstract Haploinsufficiency of the ATP6V1B2, a subunit of V‐ATPases, underlies genetic disorders including Dominant deafness‐onychodystrophy (DDOD), deafness, onychodystrophy, osteodystrophy, mental retardation and seizures (DOORS), and Zimmermann‐Laband syndromes, all characterized by congenital he...

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Main Authors: Gege Wei, Shiwei Qiu, Xue Gao, Lu Zheng, Yijin Chen, Ying Ma, Haifeng Feng, Jinyuan Yang, Guojie Dong, Huiyi Nie, Weihao Zhao, Xiaoge Li, Guangqin Wang, Wei Xiong, Pu Dai, Yongyi Yuan
Format: Article
Language:English
Published: Wiley 2025-08-01
Series:Advanced Science
Subjects:
adeno‐associated virus (AAV)
Atp6v1b2
cochlear hair cells
gene replacement
hearing loss
lysosome
Online Access:https://doi.org/10.1002/advs.202408878
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