Current AAV-mediated gene therapy in sensorineural hearing loss

被引：7

作者：

Qi, Jieyu ^{[1
,2
,3
,4
]}

Fu, Xiaolong ^{[1
]}

Zhang, Liyan ^{[1
]}

Tan, Fangzhi ^{[1
]}

Li, Nianci ^{[1
]}

Sun, Qiuhan ^{[1
]}

Hu, Xiaojie ^{[1
]}

He, Zuhong ^{[5
]}

Xia, Ming ^{[6
,7
]}

Chai, Renjie ^{[1
,2
,8
,9
,10
]}

机构：

[1] Southeast Univ, Zhongda Hosp, Adv Inst Life & Hlth, Sch Med,State Key Lab Digital Med Engn,Jiangsu Pro, Nanjing 210096, Peoples R China

[2] Beijing Inst Technol, Aerosp Ctr Hosp, Sch Life Sci, Dept Neurol, Beijing 100081, Peoples R China

[3] Beijing Inst Technol, State Key Lab Hearing & Balance Sci, Beijing 100081, Peoples R China

[4] Beijing Inst Technol, Adv Technol Res Inst, Jinan 250300, Peoples R China

[5] Wuhan Univ, Zhongnan Hosp, Dept Otorhinolaryngol Head & Neck Surg, Wuhan 430071, Peoples R China

[6] Shandong First Med Univ, Shandong Prov Hosp, Dept Otolaryngol, Jinan 250021, Peoples R China

[7] Shandong First Med Univ & Shandong Acad Med Sci, Med Sci & Technol Innovat Ctr, Jinan 250021, Peoples R China

[8] Nantong Univ, Coinnovat Ctr Neuroregenerat, Nantong 226001, Peoples R China

[9] Univ Elect Sci & Technol China, Sichuan Prov Peoples Hosp, Dept Otolaryngol Head & Neck Surg, Chengdu 610072, Peoples R China

[10] Southeast Univ, Shenzhen Res Inst, Shenzhen 518063, Peoples R China

来源：

FUNDAMENTAL RESEARCH | 2025年 / 5卷 / 01期

基金：

中国博士后科学基金; 中国国家自然科学基金;

关键词：

Hearing loss; Cochlea; Gene therapy; Adeno-associated virus; Auditory repair; ALIPOGENE TIPARVOVEC AAV1-LPLS447X; ADENOASSOCIATED VIRUS VECTORS; MOUSE MODEL; IN-VIVO; HAIR-CELL; ANTISENSE OLIGONUCLEOTIDES; VESTIBULAR FUNCTION; RESCUES HEARING; REPLACEMENT THERAPY; PACKAGING CAPACITY;

D O I：

10.1016/j.fmre.2022.08.015

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

The number of patients with hearing loss is on the rise due to congenital abnormalities, degenerative changes in old age, and acquired injuries such as virus or ototoxic drug-induced diseases. Hearing loss is a refractory and disabling disease that has serious negative effects on quality of life. The pathology of hearing loss in the inner ear is characterized by varying degrees of damage to the cochlear sensory epithelium cells (such as hair cells and supporting cells), stria vascularis (including marginal, intermediate and basal cells) and spiral ganglion neurons. Regeneration or direct repair of damaged cells in the inner ear is an effective way to treat sensorineural deafness. It is currently possible to regenerate hair cells to treat sensorineural hearing loss by FX-322, a small molecule drug in clinical trials. With the development of genetic engineering technology, gene therapy has brought a promising treatment strategy for many previously intractable diseases. Gene therapy has been regarded as a promising method in the treatment and rehabilitation of sensorineural hearing loss, and recombinant adenoassociated virus gene therapy has been widely used in fundamental research into hearing loss treatments. At present, gene therapy for hearing loss is transitioning from feasibility studies to explorations of its safety and its therapeutic potential. The present article reviews the concepts, strategies, and applications of gene therapy mediated by recombinant adeno-associated viruses in the field of hearing loss treatment.

引用

页码：192 / 202

页数：11

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