Blastocyst injection of wild type embryonic stem cells induces global corrections in mdx mice.
PLoS One
; 4(3): e4759, 2009.
Article
in En
| MEDLINE
| ID: mdl-19277212
ABSTRACT
Duchenne muscular dystrophy (DMD) is an incurable neuromuscular degenerative disease, caused by a mutation in the dystrophin gene. Mdx mice recapitulate DMD features. Here we show that injection of wild-type (WT) embryonic stem cells (ESCs) into mdx blastocysts produces mice with improved pathology and function. A small fraction of WT ESCs incorporates into the mdx mouse nonuniformly to upregulate protein levels of dystrophin in the skeletal muscle. The chimeric muscle shows reduced regeneration and restores dystrobrevin, a dystrophin-related protein, in areas with high and with low dystrophin content. WT ESC injection increases the amount of fat in the chimeras to reach WT levels. ESC injection without dystrophin does not prevent the appearance of phenotypes in the skeletal muscle or in the fat. Thus, dystrophin supplied by the ESCs reverses disease in mdx mice globally in a dose-dependent manner.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Blastocyst
/
Genetic Therapy
/
Embryonic Stem Cells
/
Muscular Dystrophy, Animal
Limits:
Animals
Language:
En
Journal:
PLoS One
Journal subject:
CIENCIA
/
MEDICINA
Year:
2009
Document type:
Article
Affiliation country:
United States