Delta-Sarcoglycan Gene Therapy Halts Progression of Cardiac Dysfunction, Improves Respiratory Failure, and Prolongs Life in Myopathic Hamsters
- Hoshijima, Masahiko
- Hayashi, Takeharu
- Jeon, Young E.
- Fu, Zhenxing
- Gu, Yusu
- Dalton, Nancy D.
- Ellisman, Mark H.
- Xiao, Xiao
- Powell, Frank L.
- Ross, John
Publication date
January 2011
DOIAbstract
The BIO14.6 hamster provides a useful model of hereditary cardiomyopathies and muscular dystrophy. Previous delta-sarcoglycan (δSG) gene therapy (GT) studies were limited to neonatal and young adult animals, and prevented the development of cardiac and skeletal muscle dysfunction. GT of a pseudo-phosphorylated mutant of phospholamban (S16EPLN) moderately alleviated the progression of cardiomyopathy
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