Research progress of gene therapy in clinical application.
- Author:
Xi CHEN
1
;
Liang CHEN
1
;
Dali LI
1
Author Information
- Publication Type:Journal Article
- Keywords: CRISPR/Cas9 system; approved drugs; clinical trails; gene therapy; genetic rare disease; genome editing technology
- MeSH: CRISPR-Cas Systems; Gene Editing; Genetic Therapy; Humans
- From: Chinese Journal of Biotechnology 2019;35(12):2295-2307
- CountryChina
- Language:Chinese
- Abstract: In the 1960s, scientists first raised the idea of curing genetic diseases using gene therapy. This new conceptual strategy aimed to achieve a much longer therapeutic effect by introducing exogenous genetic materials into the patients. After more than five decades of ups and downs, gene therapy has been brought into a new era by those milestone breakthroughs in the 21st century. Here we reviewed and summarized the history and breakthroughs of gene therapy, including some critical clinical trials, approved drugs, and emerging gene editing techniques. We believe that with their unique advantages over traditional therapies, more gene therapies will become practical approaches to genetic diseases and benefit the entire human race.
