[Progress in research on pathogenic genes and gene therapy for inherited retinal diseases]

Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2017 Feb 10;34(1):118-123. doi: 10.3760/cma.j.issn.1003-9406.2017.01.028.
[Article in 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.

Publication types

  • Review

MeSH terms

  • Biomedical Research / methods
  • Biomedical Research / trends
  • Clinical Trials as Topic
  • Genetic Predisposition to Disease / genetics*
  • Genetic Therapy / methods*
  • Genetic Therapy / trends
  • Humans
  • Mutation*
  • Retinal Diseases / genetics*
  • Retinal Diseases / therapy*
  • Treatment Outcome