Grant: Operating Grant - PA: IMHA New investigators - Bridge Funding

Muscular dystrophies are devastating and incurable inherited diseases. They are genetic disorders that weaken the muscles. There is currently no effective treatment except palliative therapy for any form of muscular dystrophy. Our research focuses on identifying molecular therapies for muscular dystrophies, especially in the promising field of antisense therapy. Antisense therapy is an approach to fighting diseases using DNA-like molecules. Our exon skipping therapy employs a cocktail of synthetic DNA-like molecules as "DNA Band-Aids" to target the mutated parts of the gene, and produce functional proteins. We will identify antisense drugs to rescue several forms of muscular dystrophies.