Background Major efforts have been made in the last decade to develop and improve
therapies for proximal spinal muscular atrophy (SMA). The introduction of …

The market for large molecule biologic drugs has grown rapidly, including antisense
oligonucleotide (ASO) drugs. ASO drugs work as single-stranded synthetic oligonucleotides …

We treated a 27-year-old patient with Duchenne's muscular dystrophy (DMD) with
recombinant adeno-associated virus (rAAV) serotype 9 containing d Sa Cas9 (ie,“dead” …

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by
the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene …

Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder that
causes debilitating muscle weakness and atrophy due to a loss of the dystrophin protein …

An N-of-1 trial was developed to deliver a dCas9-VP64 transgene designed to upregulate
the cortical dystrophin as a custom therapy for a Duchenne muscular dystrophy (DMD) …

Histone deacetylases (HDACs) are epigenetic enzymes which participate in transcriptional
repression and chromatin condensation mechanisms by removing the acetyl moiety from …

Background: Adeno-associated virus (AAV) vectors are a promising platform for in vivo
transfer of transgenes designed to treat diseases. Pre-existing humoral immunity to these …

Dystrophinopathies are x-linked muscular disorders which emerge from mutations in the
Dystrophin gene, including Duchenne and Becker muscular dystrophy, and dilated …

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease arising from loss-
of-function mutations in the dystrophin gene and characterized by progressive muscle …