Introduction Gene therapy is becoming increasingly common in clinical practice, giving hope
for the correction of a wide range of human diseases and defects. The CRISPR/Cas9 …

The emergence of the clustered regularly interspaced short palindromic repeats
(CRISPR)/CRISPR-associated protein 9 (Cas9) genome-editing system has brought about a …

Abstract Muscular Dystrophy Association (MDA) has invested over $125 M in the
development of gene therapy for neuromuscular disorders (NMD) over the past 20 years. As …

Large genes including several CRISPR-Cas modules like gene activators (CRISPRa)
require dual adeno-associated viral (AAV) vectors for an efficient in vivo delivery and …

Extracellular vesicles (EVs) mediate intercellular biomolecule exchanges in the body,
making them promising delivery vehicles for therapeutic cargo. Genetic engineering by the …

Inherited retinal diseases (IRDs) are a leading cause of blindness in industrialized countries,
and gene therapy is quickly becoming a viable option to treat this group of diseases. Gene …

Muscular dystrophies (MDs) comprise a diverse group of inherited disorders characterized
by progressive muscle loss and weakness. Given the genetic etiology underlying MDs …

Muscle-directed gene therapy with adeno-associated viral (AAV) vectors is undergoing
clinical development for treating neuromuscular disorders and for systemic delivery of …

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

CRISPR gene-editing therapies for hypertrophic cardiomyopathy | Nature Medicine Skip to
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