New advances in gene therapy with adenoviral vectors for inherited retinal diseases
10.3760/cma.j.cn511434-20190705-00214
- VernacularTitle:遗传性视网膜疾病腺相关病毒载体基因治疗新进展
- Author:
Kejiong SHEN
1
;
Yin SHEN
Author Information
1. 武汉大学人民医院眼科中心 430060
- From:
Chinese Journal of Ocular Fundus Diseases
2020;36(3):242-248
- CountryChina
- Language:Chinese
-
Abstract:
Adeno-associated viral vector (AAV) is the most important viral tool and has been widely used in gene therapy. Because of its small size, non-enveloped, non-pathogenic and other characteristics, so it is one of the main means to treat hereditary retinal diseases. Aiming at MERTK for retinitis pigmentosa, ND4 for Leber hereditary optic neuropathy or RPE1 for choroideremia, AAV gene therapy improved half patients' visual acuity in clinic tests. Besides, there are some clinic tests in progress for Leber’s congenital amaurosis, X-linked retinoschisis, Achromatopsia, age-related macular degeneration. But more researches need to be found before clinic test for Stargardt disease, Usher syndrome and nanophthalmos. At present, AAV gene therapy is mainly used for recessive hereditary retinal diseases, and technology is needed to intervene for dominant retinal diseases. For the treatment of hereditary retinal diseases, this will be an important and complex systematic project, which requires more human and material resources to participate in and study together, and we expect to have a great breakthrough in the near future.
