Gene therapy for the muscular dystrophies has evolved as a promising treatment for this
progressive group of disorders. Although corticosteroids and/or supportive treatments …

This review considers the most significant progress in developing genetic therapeutic
interventions for Duchenne muscular dystrophy (DMD), in order to illustrate the challenges …

Duchenne muscular dystrophy (DMD) is a devastating muscle disorder that affects 1 in
3,500 boys. Despite years of research and considerable progress in understanding the …

Muscular dystrophies are individually rare genetic disorders that cause much chronic
disability, affecting young children and adults. In the past 20 years, more than 30 genetic …

In the last few years, significant advances have occurred in the preclinical and clinical work
toward gene and cell therapy for muscular dystrophy. At the time of this writing, several trials …

Clinical trials represent a critical avenue for new treatment development, where early
phases (I, I/II) are designed to test safety and effectiveness of new therapeutics or diagnostic …

This review updates the state‐of‐the art accomplishments of the multifaceted gene‐based
therapies, which include DNA or RNA as either therapeutic tools or targets for the treatment …

Gene therapy for muscular dystrophy represents a promising avenue of pursuit for a disease
with a limited repertoire of treatment. Recent successes in the research arena using adeno …

These studies should provide proof of principle of these experimental approaches in
humans. It is likely that further refinement will be required before extending their use to the …

Duchenne muscular dystrophy (DMD) is a recessive lethal inherited muscular dystrophy
caused by mutations in the gene encoding dystrophin, a protein required for muscle fibre …