Research progress on disease models and gene therapy of Duchenne muscular dystrophy / 浙江大学学报·医学版
Journal of Zhejiang University. Medical sciences
;
(6): 648-654, 2016.
Artigo
em Chinês
| WPRIM
| ID: wpr-300833
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.
Texto completo:
DisponíveL
Índice:
WPRIM (Pacífico Ocidental)
Assunto principal:
Terapêutica
/
Terapia Genética
/
Distrofina
/
Camundongos Endogâmicos mdx
/
Distrofia Muscular de Duchenne
/
Modelos Animais de Doenças
/
Células-Tronco Pluripotentes Induzidas
/
Genética
Limite:
Animais
/
Humanos
Idioma:
Chinês
Revista:
Journal of Zhejiang University. Medical sciences
Ano de publicação:
2016
Tipo de documento:
Artigo
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