Research progress on disease models and gene therapy of Duchenne muscular dystrophy.
- Author:
Tongyu LI
1
,
2
,
3
,
4
;
Ping LIANG
1
;
Author Information
1. Division of Hepatobilitary and Pancreatic Surgery, the First Affiliated Hospital, Zhejiang University School of Medicine
2. Key Laboratory of Combined Multi-organ Transplantation, Ministry of Public Health
3. Key Laboratory of Organ Transplantation, Zhejiang Province, Hangzhou 310003, China
4. Institute of Translational Medicine, Zhejiang University, Hangzhou 310029, China.
- Publication Type:Journal Article
- MeSH:
Animals;
Disease Models, Animal;
Dystrophin;
genetics;
Genetic Therapy;
trends;
Humans;
Induced Pluripotent Stem Cells;
Mice;
Mice, Inbred mdx;
genetics;
Muscular Dystrophy, Duchenne;
genetics;
therapy
- From:
Journal of Zhejiang University. Medical sciences
2016;45(6):648-654
- CountryChina
- Language:Chinese
-
Abstract:
Duchenne muscular dystrophy (DMD) is an X-linked, recessive and lethal genetic disease, which usually caused by gene mutations and the underlying mechanisms are complicated and diverse. The causal gene of DMD is the largest one in human that locates in the region of Xp21.2, encoding dystrophin. Currently there is no effective treatment for DMD patients. The treatment of DMD depends on gene mutation and molecular mechanism study of the disease, which requires reliable disease models such as mdx mouse model. Recently, researchers have increasingly discovered gene therapy strategies for DMD, and the efficacy has been demonstrated in DMD animal models. In addition, induced pluripotent stem cell technology can provide patient-specific cell source, offering a new platform for mechanism and therapy study of DMD.
