The mammalian heart is responsible for not only pumping blood throughout the body but
also adjusting this pumping activity quickly depending upon sudden changes in the …

Muscular dystrophies are a heterogeneous group of genetic disorders characterized by
muscle weakness and wasting. Duchenne muscular dystrophy (DMD) is the most common …

Adeno-associated viruses (AAVs) are small, nonenveloped single-stranded DNA viruses
that require helper viruses to facilitate efficient replication. Despite the presence of humoral …

Delivering and expressing a gene of interest in cells or living animals has become a pivotal
technique in biomedical research and gene therapy. Among viral delivery systems, adeno …

Gene therapies using adeno-associated viral (AAV) vectors have advanced into clinical
trials for several diseases, including Duchenne muscular dystrophy (DMD). A limitation of …

Muscle represents an important tissue target for adeno-associated viral (AAV) vector-
mediated gene transfer of the factor IX (FIX) gene in hemophilia B (HB) subjects with …

The ability to specifically, safely, and efficiently transfer therapeutic payloads to the striated
musculature via a minimally invasive delivery route remains one of the most important but …

Despite an expansive wealth of research following the discovery of the DMD gene 25 years
ago, there is still no curative treatment for Duchenne muscular dystrophy. However, there …

Duchenne muscular dystrophy (DMD) is a fatal, X-linked muscle disease caused by
mutations in the dystrophin gene. Adeno-associated viral (AAV) vector-mediated gene …

Adeno-associated virus (AAV) vectors are promising candidates for gene therapy and have
been explored as gene delivery vehicles in the treatment of Duchenne Muscular Dystrophy …