Duplication 2 Research for Duchenne Muscular Dystrophy
A new form of dystrophin discovered at Nationwide Children’s Hospital suggests a route to therapy for patients with mutations in the first five exons of the dystrophin gene.
Dr. Kevin Flanigan’s team began by studying exon skipping as a potential therapy for duplicated exons. What began as a novel therapy for skipping the most common duplication – exon 2 – led him and his team to uncover a novel element in the gene that creates a shorter, highly functional version of the dystrophin protein.
Learn more at www.CureDuchenne.org/Flanigan