Cardiomyopathy

Partial-heart dystrophin gene transfer may correct hereditary heart disease

Published in Gene Therapy Weekly, September 16th, 2004

Partial-heart dystrophin gene transfer may correct hereditary heart disease.

According to a study from the United States, "gene therapy holds great promise for curing Duchenne muscular dystrophy (DMD), the most common fatal inherited childhood muscle disease. Success of DMD gene therapy depends upon functional improvement in both skeletal and cardiac muscle. Numerous gene transfer studies have been performed to correct skeletal muscle pathology, yet little is known about cardiomyopathy gene therapy."

"Since complete transduction of the entire heart is an impractical goal, it becomes critical to determine the minimal level of correction needed for...

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Source: Gene Therapy Weekly (2004-09-16)

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