DMD Gene Therapy Resource Center

Gene therapy represents the vanguard for treating the world’s most deadly genetic diseases. Duchenne muscular dystrophy (DMD) is among a growing list of rare diseases where gene therapies are either approved for use by agencies such as the FDA and EMA or making their way through the late-stage clinical pipeline. Rare Disease Advisor’s DMD gene therapy resource center offers a range of news, insights, and resources on the research and development of DMD gene therapies.

Be sure to check back regularly as RDA continues to cover gene therapy news for the treatment of DMD.

DNA strand

Examining Exon Skipping as a Gene Therapy Approach in DMD

Research on gene therapy has become increasingly prominent in recent years, and the trajectory is expected to continue. Since the Human Genome Project was completed a few decades ago, the collective imagination of scientists and physicians worldwide has been ignited by the possibilities that gene therapy opens up.  Gene therapy offers the tantalizing prospect of…


DNA gel close-up

Prime Editing Could Be Used to Correct DMD-Causing Point Mutations, Study Says

Prime editing allows the introduction of specific mutations in the DMD gene, a new study published in the International Journal of Molecular Sciences reported.  Based on this finding, the authors of the study concluded that prime editing could therefore be used to correct point mutations in the DMD gene causing Duchenne muscular dystrophy (DMD).  For…
Chromosome DNA

Novel CRISPR/Cas System May Efficiently Repair Mutations Causing DMD and CF

A novel clustered regularly interspaced short palindromic repeats (CRISPR)/Cas-based technology developed by Script Biosciences can correct mutations causing Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF) with high efficiency and low on-target DNA damage rate, according to a study presented at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. According to…


Jerry Mendell, MD, FAAN, Nationwide Children’s Hospital & Ohio State University, Columbus, OH, comments on the use of gene therapy in patients with Duchenne muscular dystrophy (DMD).

Clinical Trials

Read about the latest experimental therapies for DMD

Recent Studies

Dr. Eric Olson Discusses the Use of Gene Therapy in the Treatment of DMD

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Eric Olson, PhD, chief scientific advisor at Vertex Pharmaceuticals and founding chair of the molecular biology department at the University of Texas-Southwestern Medical Center. Dr. Olson is an expert on Duchenne muscular dystrophy and CRISPR gene editing technology.