Open AccessRecent development of AAV-based gene therapies for inner ear disorders
Author(s) -
Yiyang Lan,
Yong Tao,
Yunfeng Wang,
Junzi Ke,
Qiuxiang Yang,
Xiaoyi Liu,
Bing Su,
Yi-Chien Wu,
Chao-Po Lin,
Guisheng Zhong
Publication year - 2020
Publication title -
gene therapy
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.332
H-Index - 159
eISSN - 1476-5462
pISSN - 0969-7128
DOI - 10.1038/s41434-020-0155-7
Subject(s) - genetic enhancement , biology , viral vector , cochlea , vector (molecular biology) , animal model , gene transfer , hearing loss , inner ear , computational biology , gene delivery , neuroscience , gene , bioinformatics , medicine , genetics , audiology , recombinant dna , endocrinology
Gene therapy for auditory diseases is gradually maturing. Recent progress in gene therapy treatments for genetic and acquired hearing loss has demonstrated the feasibility in animal models. However, a number of hurdles, such as lack of safe viral vector with high efficiency and specificity, robust deafness large animal models, translating animal studies to clinic etc., still remain to be solved. It is necessary to overcome these challenges in order to effectively recover auditory function in human patients. Here, we review the progress made in our group, especially our efforts to make more effective and cell type-specific viral vectors for targeting cochlea cells.