Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne muscular dystrophy (DMD). The study, recently published in Nature Communications , demonstrates the effectiveness of their novel gene therapy technology in improving muscle tissue and overall strength in mice models with Duchenne muscular dystrophy. Duchenne muscular dystrophy is a genetic disorder caused by mutations in the DMD gene, resulting in a lack of the protein dystrophin.
This deficiency leads to progressive muscle weakness and loss of muscle tissue over time. Patients with the disease experience impaired mobility, heart and lung problems, and ultimately a shortened life expectancy. Current gene therapy for Duchenne muscular dystrophy utilizes a truncated version of dystrophin.
Unfortunately, this option doesn't fully protect the muscles because it lacks many important functional domains of full-length dystrophin." Renzhi Han, PhD, senior author of the study and professor of pediatrics at the IU School of Medicine While the U.S.
Food and Drug Administration recently approved a micro-dystrophin gene therapy for Duchenne muscular dystrophy, Han said the therapeutic outcomes have been less satisfactory than expected. Building on their experience using adeno-associated virus methods to deliver extra-large therapeutic genes into cells, Han a.