国际眼科纵览 ›› 2022, Vol. 46 ›› Issue (6): 565-571.doi: 10.3760/ cma.j.issn.1673-5803.2022.06.016

• 综述 • 上一篇    下一篇

腺相关病毒介导的角膜疾病基因治疗基础研究进展

王烽1  赖江峰1  邵毅2   

  1. 1广东省梅州市人民医院眼科 514000;2南昌大学第一附属医院眼科 330006
  • 收稿日期:2022-02-05 出版日期:2022-12-22 发布日期:2022-12-22
  • 通讯作者: 邵毅,Email:freebee99@163.com
  • 基金资助:
    国家自然科学基金(82160195);中央引导地方科技发展资金(20211ZDG02003);江西省重点研发项目(20181BBG700004,20203BBG73059);广东省梅州市人民医院科研培育项目(PY-C2021060)

Advances in basic research on gene therapy of corneal diseases mediated by adeno-associated virus

Wang Feng1, Lai Jiangfeng1, Shao Yi2   

  1. 1Department of Ophthalmology, Meizhou Pepole’s Hospital, Meizhou, Guangdong 514000, China; 2Department of Ophthalmology, the First Affiliated Hospital of Nanchang University, Nanchang 330006, China
  • Received:2022-02-05 Online:2022-12-22 Published:2022-12-22
  • Contact: Shao Yi, Email: freebee99 @163.com
  • Supported by:
    National Natural Science Foundation (82160195); Central Government Guides Local Science and Technology Development Foundation (20211ZDG02003); Key Research Foundation of Jiangxi Province (20181BBG70004, 20203BBG73059); Research and cultivation project of Guangdong Meizhou People's Hospital (PY-C2021060)

摘要: 腺相关病毒(adeno associated virus,AAV)介导的基因治疗通过基因增强、基因删除和/或基因编辑将遗传物质靶向角膜疾病,靶向给药的易可及性和高转导效率使得AAV介导的基因治疗可有效治疗角膜疾病,包括抑制角膜新生血管形成、改善与粘多糖沉积症相关的角膜混浊、促进角膜机械损伤和化学损伤的愈合、治疗感染性角膜炎、有效地转染角膜内皮细胞治疗先天性遗传性角膜内皮营养不良,为治疗角膜疾病提供了新的途径。(国际眼科纵览,2022, 46:565-571)

关键词: 腺相关病毒, 角膜, 角膜疾病, 基因治疗

Abstract: Adeno Associated Virus (AAV) mediated gene therapy targets genetic material to corneal diseases through gene enhancement, gene deletion and/or gene editing. The accessibility of targeted drug delivery and high transduction efficiency enable AAV mediated gene therapy to effectively treat corneal diseases, including inhibiting corneal neovascularization and improving corneal opacity related to mucopolysaccharosis. To promote the healing of corneal mechanical and chemical injuries, treat infectious keratitis, and effectively transfect corneal endothelial cells to treat congenital hereditary corneal endothelial dystrophy, it provide a new way to treat corneal diseases. (Int Rev Ophthalmol, 2022, 46: 565-571)

Key words: adeno-associated viruses, cornea, corneal diseases,  , gene therapy