Gene therapy development in hearing research in China

作者: Zhen Zhang , Jiping Wang , Chunyan Li , Wenyue Xue , Yazhi Xing

DOI: 10.1038/S41434-020-0177-1

关键词: Animal modelBasic researchGenetic enhancementNeuroscienceBiologyAuditory functionHearing researchNoise exposureSensorineural hearing loss

摘要: Sensorineural hearing loss, the most common form of impairment, is mainly attributable to genetic mutations or acquired factors, such as aging, noise exposure, and ototoxic drugs. In field gene therapy, advances in physiological studies profound increases knowledge regarding underlying mechanisms have yielded great progress terms restoring auditory function animal models deafness. Nonetheless, many challenges associated with translation from basic research clinical therapies remain be overcome before a total restoration can expected. recent years, Chinese teams promoted various developmental efforts this field, including sequencing identify additional potential loci that cause deafness, elucidate molecular mechanisms, optimize vectors delivery routes. review, we summarize state focus on therapy model optimization therapeutic strategies China.

参考文章(132)
Xi Gu, Renjie Chai, Luo Guo, Biao Dong, Wenyan Li, Yilai Shu, Xinsheng Huang, Huawei Li, Transduction of Adeno-Associated Virus Vectors Targeting Hair Cells and Supporting Cells in the Neonatal Mouse Cochlea. Frontiers in Cellular Neuroscience. ,vol. 13, pp. 8- ,(2019) , 10.3389/FNCEL.2019.00008
Yongli Zhang, Qi Tang, Ruoyan Xue, Juanjuan Gao, Hua Yang, Zhiqiang Gao, Gan Lin, Absence of Atoh1 induced partially different cell fates of cochlear and vestibular sensory epithelial cells in mice. Acta Oto-laryngologica. ,vol. 138, pp. 972- 976 ,(2018) , 10.1080/00016489.2018.1497855
Kevin Isgrig, Devin S. McDougald, Jianliang Zhu, Hong Jun Wang, Jean Bennett, Wade W. Chien, AAV2.7m8 is a powerful viral vector for inner ear gene therapy Nature Communications. ,vol. 10, pp. 427- ,(2019) , 10.1038/S41467-018-08243-1
Omar Akil, Frank Dyka, Charlotte Calvet, Alice Emptoz, Ghizlene Lahlou, Sylvie Nouaille, Jacques Boutet de Monvel, Jean-Pierre Hardelin, William W. Hauswirth, Paul Avan, Christine Petit, Saaid Safieddine, Lawrence R. Lustig, Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model. Proceedings of the National Academy of Sciences of the United States of America. ,vol. 116, pp. 4496- 4501 ,(2019) , 10.1073/PNAS.1817537116
Bence György, Carl Nist-Lund, Bifeng Pan, Yukako Asai, K. Domenica Karavitaki, Benjamin P. Kleinstiver, Sara P. Garcia, Mikołaj P. Zaborowski, Paola Solanes, Sofia Spataro, Bernard L. Schneider, J. Keith Joung, Gwenaëlle S. G. Géléoc, Jeffrey R. Holt, David P. Corey, Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss Nature Medicine. ,vol. 25, pp. 1123- 1130 ,(2019) , 10.1038/S41591-019-0500-9
Wenjun Xia, Jiongjiong Hu, Jing Ma, Jianbo Huang, Xu Wang, Nan Jiang, Jin Zhang, Zhaoxin Ma, Duan Ma, Novel TRRAP mutation causes autosomal dominant non-syndromic hearing loss. Clinical Genetics. ,vol. 96, pp. 300- 308 ,(2019) , 10.1111/CGE.13590
Fangzhi Tan, Cenfeng Chu, Jieyu Qi, Wenyan Li, Dan You, Ke Li, Xin Chen, Weidong Zhao, Cheng Cheng, Xiaoyi Liu, Yunbo Qiao, Bing Su, Shuijin He, Chao Zhong, Huawei Li, Renjie Chai, Guisheng Zhong, AAV-ie enables safe and efficient gene transfer to inner ear cells Nature Communications. ,vol. 10, pp. 3733- 3733 ,(2019) , 10.1038/S41467-019-11687-8
Shasha Zhang, Yuan Zhang, Ying Dong, Lingna Guo, Zhong Zhang, Buwei Shao, Jieyu Qi, Han Zhou, Weijie Zhu, Xiaoqian Yan, Guodong Hong, Liyan Zhang, Xiaoli Zhang, Mingliang Tang, Chunjie Zhao, Xia Gao, Renjie Chai, Knockdown of Foxg1 in supporting cells increases the trans-differentiation of supporting cells into hair cells in the neonatal mouse cochlea Cellular and Molecular Life Sciences. ,vol. 77, pp. 1401- 1419 ,(2020) , 10.1007/S00018-019-03291-2
Xiao-Jun Ji, Wei Chen, Xiao Wang, Yue Zhang, Qian Liu, Wei-Wei Guo, Jian-Guo Zhao, Shi-Ming Yang, Canalostomy is an ideal surgery route for inner ear gene delivery in big animal model. Acta Oto-laryngologica. ,vol. 139, pp. 939- 947 ,(2019) , 10.1080/00016489.2019.1654130
Cheng Cheng, Yunfeng Wang, Luo Guo, Xiaoling Lu, Weijie Zhu, Waqas Muhammad, Liyan Zhang, Ling Lu, Junyan Gao, Mingliang Tang, Fangyi Chen, Xia Gao, Huawei Li, Renjie Chai, Age-related transcriptome changes in Sox2+ supporting cells in the mouse cochlea Stem Cell Research & Therapy. ,vol. 10, pp. 365- ,(2019) , 10.1186/S13287-019-1437-0