Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

Importance Micro-dystrophin gene transfer shows promise for treating patients with
Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype …

Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …

Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …

Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000
male births and caused by mutations in the dystrophin gene. Genome editing has the …

Duchenne muscular dystrophy (DMD) is an X-linked, muscle wasting disease that affects 1
in 5000 males. Affected individuals become wheelchair bound by the age of twelve and …

Dystrophin is a long rod-shaped protein that connects the subsarcolemmal cytoskeleton to a
complex of proteins in the surface membrane (dystrophin protein complex, DPC), with further …