Duchenne Muscular Dystrophy Cardiac Tissue Protection

Researchers have engineered a modified dystrophin gene utilized to protect cardiac tissue. A specific domain has been identified within the dystrophin gene to have cardiac tissue protection. This specific domain has been reduced to essential components to allow cellular infiltration. This region has shown a significant recruitment of cavin-1, a cardiac specific dystrophin binding protein.

BACKGROUND:
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder that results in deficient activity of dystrophin. Dystrophin is a protein that links the cytoskeleton to the extracellular matrix. DMD usually detected in boys aged 2-5 years. Without treatment death usually occurs at about 20 years old. Within cardiac tissue, cardiomyopathy and/or cardiac arrhythmia tends to occur. Approximately 90% of patients have subclinical or clinical cardiac involvement. Death from DMD usually occurs due to cardiac or respiratory failure.

Not much is known about DMD progression in cardiac tissue and how to protect tissue from DMD. Even more, a cardiac tissue dystrophin protection domain has yet to be discovered. This technology has identified a specific domain important for cardiac tissue protection.

APPLICATIONS:
Duchenne muscular dystrophy cardiac tissue protection

ADVANTAGES:
Preventative measure for DMD patients with cardiac tissue complications