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AAV 载体基因治疗内耳疾病的最新进展。

Recent development of AAV-based gene therapies for inner ear disorders.

机构信息

iHuman Institute, ShanghaiTech University, Shanghai, 201210, China.

School of Life Science and Technology, ShanghaiTech University, Shanghai, 201210, China.

出版信息

Gene Ther. 2020 Aug;27(7-8):329-337. doi: 10.1038/s41434-020-0155-7. Epub 2020 May 18.

Abstract

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.

摘要

基因治疗在听觉疾病方面正逐渐成熟。基因治疗对遗传性和获得性听力损失的治疗最近取得了进展,在动物模型中证明了其可行性。然而,仍有许多障碍需要克服,例如缺乏高效且具有特异性的安全病毒载体、稳健的耳聋大型动物模型、将动物研究转化为临床等。为了有效地恢复人类患者的听觉功能,必须克服这些挑战。在这里,我们综述了我们小组所取得的进展,特别是我们努力使针对耳蜗细胞的更有效和细胞类型特异性的病毒载体。

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