Gene therapy development in hearing research in China
Gene therapy development in hearing research in China
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中国听力研究中的基因治疗进展
DOI：
10.1038/s41434-020-0177-1
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发表时间：
2020-07
期刊：
影响因子：
5.1
通讯作者：
Liu Feng
中科院分区：
文献类型：
--
作者：
Zhang Zhen;Wang Jiping;Li Chunyan;Xue Wenyue;Xing Yazhi;Liu Feng
感音神经性听力损失是最常见的听力损害形式，主要归因于基因突变或后天因素，如衰老、噪声暴露和耳毒性药物。在基因治疗领域，基因和生理学研究的进展以及对潜在机制的认识的深刻增加在耳聋动物模型中恢复听觉功能方面取得了巨大进展。尽管如此，在预期听觉功能完全恢复之前，与从基础研究到临床治疗的转化相关的许多挑战仍有待克服。近年来，中国研究团队在这一领域开展了各种开发工作，包括基因测序以确定导致耳聋的其他潜在位点，阐明潜在分子机制的研究以及优化载体和传递途径的研究。本文就基因治疗的动物模型研究进展及我国基因治疗策略的优化进行综述。
Sensorineural hearing loss, the most common form of hearing impairment, is mainly attributable to genetic mutations or acquired factors, such as aging, noise exposure, and ototoxic drugs. In the field of gene therapy, advances in genetic and physiological studies and profound increases in knowledge regarding the underlying mechanisms have yielded great progress in terms of restoring the auditory function in animal models of deafness. Nonetheless, many challenges associated with the translation from basic research to clinical therapies remain to be overcome before a total restoration of auditory function can be expected. In recent years, Chinese research teams have promoted various developmental efforts in this field, including gene sequencing to identify additional potential loci that cause deafness, studies to elucidate the underlying molecular mechanisms, and research to optimize vectors and delivery routes. In this review, we summarize the state of the field and focus mainly on the progress of gene therapy in animal model studies and the optimization of therapeutic strategies in China.
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