Gene Therapy for Muscular Dystrophies: Progress and Challenges

Gene Therapy for Muscular Dystrophies: Progress and Challenges

Muscular dystrophies are groups of inherited progressive diseases of the muscle caused by mutations of diverse genes related to normal muscle function. Although there is no current effective treatment for these devastating diseases, various molecular strategies have been developed to restore the expressions of the associated defective proteins. In preclinical animal models, both viral and nonvi...

Advances in gene therapy for muscular dystrophies

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 integrity. So far, many approaches have been tested from the traditional gene addition to newer advanced approaches based on manipulation of the cellular machinery either at the gene transcription, mRNA processing or transl...

Progress and prospects of gene therapy clinical trials for the muscular dystrophies.

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 indicators. A number of recent advances have spurred renewed optimism toward initiating clinical trials and developing refined therapies for the muscular dystrophies (MD's) and other myogenic disorders. MD's encom...

Gene Therapy: Progress and Challenges

Over the past decade, success in the treatment of serious genetic disorders via gene therapy was finally achieved. However, this progress was tempered by the occurrence of serious adverse events related to vector integration into the genome and activation of adjacent proto-oncogenes. Investigators are now focused on retaining the clinical potential of integrating vectors while decreasing the ri...

Progress and Challenges in AAV-Mediated Gene Therapy for Duchenne Muscular Dystrophy