Progress in research on pathogenic genes and gene therapy for inherited retinal diseases.
10.3760/cma.j.issn.1003-9406.2017.01.028
- Author:
Ling ZHU
1
;
Cong CAO
;
Jiji SUN
;
Tao GAO
;
Xiaoyang LIANG
;
Zhipeng NIE
;
Yanchun JI
;
Pingping JIANG
;
Minxin GUAN
Author Information
1. Institute of Genetics, Zhejiang University School of Medicine, Hangzhou, Zhejiang 310058, China. ppjiang@zju.edu.cn.
- Publication Type:Journal Article
- MeSH:
Biomedical Research;
methods;
trends;
Clinical Trials as Topic;
Genetic Predisposition to Disease;
genetics;
Genetic Therapy;
methods;
trends;
Humans;
Mutation;
Retinal Diseases;
genetics;
therapy;
Treatment Outcome
- From:
Chinese Journal of Medical Genetics
2017;34(1):118-123
- CountryChina
- Language:Chinese
-
Abstract:
Inherited retinal diseases (IRDs), including retinitis pigmentosa, Usher syndrome, Cone-Rod degenerations, inherited macular dystrophy, Leber's congenital amaurosis, Leber's hereditary optic neuropathy are the most common and severe types of hereditary ocular diseases. So far more than 200 pathogenic genes have been identified. With the growing knowledge of the genetics and mechanisms of IRDs, a number of gene therapeutic strategies have been developed in the laboratory or even entered clinical trials. Here the progress of IRD research on the pathogenic genes and therapeutic strategies, particularly gene therapy, are reviewed.
