Basic and clinical research progress of AAV-based gene therapy <?A3B2 pi6?>for hereditary deafness
<p indent="0mm">Hearing impairment has been widely concerned as a social health problem with high incidence. About 60% of cases of sensorineural hearing loss are caused by mutations in genetic material. The clinical treatment of hereditary deafness mainly relies on cochlear implants and hearing AIDS, and the development of more effective and comprehensive biological treatment methods is imminent. Repairing the damaged genes is an effective way to fundamentally treat hereditary deafness. Several studies have shown that the replacement or repair of mutant genes in inherited deafness mouse models can effectively restore the impaired hearing in mice. At present, gene therapy has successfully restored hearing in patients with hereditary deafness carrying <italic>OTOF</italic> mutations. There are more than 200 deafness genes including <italic>OTOF</italic>. As a precision therapy, there are still many problems need to be solved in the application of gene therapy in hereditary deafness. In this paper, the occurrence and development of hereditary deafness are summarized, and then the current research progress of gene therapy applied to the clinical treatment of hereditary deafness is deeply discussed, and the dilemma and challenge of gene therapy for deafness at this stage are put forward.
Authors
- Jieyu Qi (ORCID: https://orcid.org/0000-0001-8780-0710)
- Xuehan Yang
- Yinyi Zhou
- Fangzhi Tan (ORCID: https://orcid.org/0000-0002-8230-1784)
- Renjie Chai (ORCID: https://orcid.org/0000-0002-3885-543X)
- Xinru Zhang
- Xianmin Wu
- Xiaohan Wang (ORCID: https://orcid.org/0000-0003-3027-7265)
- Liyan Zhang (ORCID: https://orcid.org/0000-0001-8410-9262)
- Xiangyan Chen (ORCID: https://orcid.org/0000-0003-2002-0327)
- Jinyi Fan
- Yicheng Lu
Publication Details
- Journal
- Scientia Sinica Vitae
- Published
- 2024-11-20
- DOI
- https://doi.org/10.1360/ssv-2024-0031
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 0.00