Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

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 …

The success of base editors for the study and treatment of genetic diseases depends on the
ability to deliver them in vivo to the relevant cell types. Delivery via adeno-associated viruses …

Adenine base editors (ABEs) composed of an engineered adenine deaminase and the
Streptococcus pyogenes Cas9 nickase enable adenine-to-guanine (A-to-G) single …

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 …

Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber
integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing …

Objective To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression
in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon …

The dystrophin complex stabilizes the plasma membrane of striated muscle cells. Loss of
function mutations in the genes encoding dystrophin, or the associated proteins, triggers …

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

The CRISPR/Cas9 genome-editing platform is a promising technology to correct the genetic
basis of hereditary diseases. The versatility, efficiency and multiplexing capabilities of the …