562. Ex Vivo Gene Therapy for Duchenne Muscular Dystrophy: Lentiviral Vectors, Exon Skipping and PhiC31 Integrase Approaches

Exon skipping therapy for Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual...

AON-Mediated Exon Skipping for Duchenne Muscular Dystrophy

Less is more: therapeutic exon skipping for Duchenne muscular dystrophy.

www.thelancet.com/neurology Vol 8 October 2009 873 Duchenne muscular dystrophy (DMD) is a lethal Xlinked progressive muscle-wasting disease caused by premature truncation of the translation of DMD mRNA into dystrophin. Owing to improved respiratory support and treatment with steroids, patients can now survive into early adulthood, which is one or two decades longer than they could survive witho...

Duchenne Muscular Dystrophy: It is time to in vivo test TEST (Targeted-Exon-Skipping Therapy)

Awaiting more successful strategies of gene therapy, longer viability of dystrophic muscle is a major goal in Duchenne Muscular Dystrophies (DMD). An alternative approach to insertion of mini-genes is targeted-exon-skipping therapy to obtain shrunk dystrophin by antisense oligonucleotides against "splicing enhancer sequences", which in vitro selectively promote Becker-like Dystrophin mRNA and p...

P164: Adeno-Associated Viral Vectors in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (BMD) is an inherited X-link disease. The incidence of this muscle-wasting disease is 1:5000 male live births. Mutation in the gene coding for dystrophin is the main cause of BMD. Most cases of this disease succumb to respiratory and cardiac failure in 3rd to 4th decades. The slow progression of BMD and recent achievement of gene therapies make it as an appropriate c...