Newswise — INDIANAPOLIS - Indiana University School of Medicine researchers have made a significant breakthrough in develop ing 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, r ecently published in Nature Communications , demonstrate s the effectiveness of their novel gene therapy technology in improv ing 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, ” said Renzhi Han, PhD , senior author of the study and professor of pediatrics at the IU School of Medicine .
“ Unfortunately, this option doesn’t fully protect the muscles because it lacks many important functional domains of full-length dystrophin." While t he 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.