Duchenne muscular dystrophy (DMD) is an X-linked recessive, infancy-onset
neuromuscular disorder characterized by progressive muscle weakness and atrophy …

Neurological disorders–disorders of the brain, spine and associated nerves–are a leading
contributor to global disease burden with a shockingly large associated economic cost …

We have shown previously that transgene expression can be suppressed in hematopoietic
cells using vectors that are responsive to microRNA (miRNA) regulation. Here we …

Preclinical or clinical trials for muscular dystrophies have met with modest success, mainly
because of inefficient delivery of viral vectors or donor cells to dystrophic muscles. We report …

A major obstacle limiting gene therapy for diseases of the heart and skeletal muscles is an
inability to deliver genes systemically to muscles of an adult organism. Systemic gene …

As a target for gene therapy, Duchenne muscular dystrophy (DMD) presents many obstacles
but also an unparalleled prospect for correction by alternative splicing. The majority of …

RNA-mediated pathogenesis is a recently developed disease model that proposes that
certain types of mutant genes produce toxic transcripts that inhibit the activities of specific …

Antisense oligonucleotide-mediated alternative splicing has great potential for treatment of
Duchenne muscular dystrophy (DMD) caused by mutations within nonessential regions of …

Since the initial characterization of the genetic defect for Duchenne muscular dystrophy,
much effort has been expended in attempts to develop a therapy for this devastating …

Systemic delivery of recombinant adeno-associated virus (rAAV) 6 vectors mediates efficient
transduction of the entire striated musculature, making this an attractive strategy for muscle …