In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

Stem cells (SCs) maintain tissue homeostasis and repair wounds. Despite marked variation
in tissue architecture and regenerative demands, SCs often follow similar paradigms in …

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 an X-linked, muscle wasting disease that affects 1
in 5000 males. Affected individuals become wheelchair bound by the age of twelve and …

Neuromuscular disorders encompass a heterogeneous group of conditions that impair the
function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being …

Cardiovascular disease remains the leading cause of morbidity and mortality in the
developed world. In recent decades, extraordinary effort has been devoted to defining the …

Recent advances in base editing have created an exciting opportunity to precisely correct
disease-causing mutations. However, the large size of base editors and their inherited off …

Recent advances in gene editing technologies are enabling the potential correction of
devastating monogenic disorders through elimination of underlying genetic mutations …

Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder with
a prevalence of approximately 1 in 3500–5000 males. DMD manifests as childhood-onset …