Rationale: Duchenne muscular dystrophy is a severe inherited form of muscular dystrophy
caused by mutations in the reading frame of the dystrophin gene disrupting its protein …

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 a severe, progressive muscle disease caused by
mutations in the dystrophin gene. The majority of DMD mutations are deletions that …

Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is
characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete …

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 …

Background: Loss of dystrophin protein causes Duchenne muscular dystrophy (DMD),
characterized by progressive degeneration of cardiac and skeletal muscles, and mortality in …

Duchenne muscular dystrophy (DMD), one of the most common neuromuscular disorders of
children, is caused by the absence of dystrophin protein in striated muscle. Deletions of …

Targeted gene-editing strategies have emerged as promising therapeutic approaches for
the permanent treatment of inherited genetic diseases. However, precise gene correction …

Duchenne muscular dystrophy is one of the most common inherited genetic diseases and is
caused by mutations to the DMD gene that encodes the dystrophin protein. Recent …

Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by mutations in the
dystrophin gene. CRISPR/Cas9 genome editing has been used to correct DMD mutations in …