Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene. Previously,
we showed that adenine base editing (ABE) can efficiently correct a nonsense point …

Muscle atrophy is the loss of skeletal muscle mass and strength in response to diverse
catabolic stimuli. At present, no effective treatments except exercise have been shown to …

Dilated cardiomyopathy (DCM) is characterized by LV (left ventricular) or biventricular
systolic dysfunction and dilation, not explained by abnormal loading conditions or coronary …

CRISPR technologies have progressed by leaps and bounds over the past decade, not only
having a transformative effect on biomedical research but also yielding new therapies that …

CRISPR editing of muscle stem cells (MuSCs) with adeno-associated virus serotype-9
(AAV9) holds promise for sustained gene repair therapy for muscular dystrophies. However …

Previous studies from others and us have demonstrated that CRISPR genome editing could
offer a promising therapeutic strategy to restore dystrophin expression and function in the …

Introduction Exon skipping compounds restoring the dystrophin transcript reading frame
have received regulatory approval for Duchenne muscular dystrophy (DMD). Recently, focus …

Background and purpose: Duchenne muscular dystrophy (DMD), a lethal X-linked recessive
muscle dystrophy, is resulted in by different mutations including mostly frame-shifting gross …

During the last decade, targeted genome-editing technologies, especially clustered
regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas) …

CRISPR is a nuclease guidance system that enables rapid and efficient gene editing of
specific DNA sequences within genomes. We review applications of CRISPR for the study …