Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused by mutations in the
dystrophin gene. To enable the non-invasive analysis of DMD gene correction strategies in …

Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …

Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …

Duchenne muscular dystrophy (DMD) is an inherited X-linked disease caused by mutations
in the gene encoding dystrophin, a protein required for muscle fiber integrity. DMD is …

Muscle stem cells (MuSCs) hold great therapeutic potential for muscle genetic disorders,
such as Duchenne muscular dystrophy (DMD). The CRISP/Cas9-based genome editing is a …

In the past decade, the development of two innovative technologies, namely, induced
pluripotent stem cells (iPSCs) and the CRISPR Cas9 system, has enabled researchers to …

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive disease of progressive
muscle weakness and wasting caused by the absence of dystrophin protein. Current gene …

The ability to efficiently modify the genome using CRISPR technology has rapidly
revolutionized biology and genetics and will soon transform medicine. Duchenne muscular …

Mutations disrupting the reading frame of the~ 2.4 Mb dystrophin-encoding DMD gene
cause a fatal X-linked muscle-wasting disorder called Duchenne muscular dystrophy (DMD) …

The CRISPR/Cas9 system is a great revolution in biology. This technology allows the
modification of genes in vitro and in vivo in a wide variety of living organisms. In most …