Genome editing, which involves the precise manipulation of cellular DNA sequences to alter
cell fates and organism traits, has the potential to both improve our understanding of human …

Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

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 monogenic disorder and a candidate for
therapeutic genome editing. There have been several recent reports of genome editing in …

Clustered regularly interspaced short palindromic repeats (CRISPR)–CRISPR associated
protein 9 (Cas9)-based therapeutics, especially those that can correct gene mutations via …

Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

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 …

With the recent development of CRISPR technology, it is becoming increasingly easy to
engineer the genome. Genome-editing systems based on CRISPR, as well as transcription …

CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic
diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in …

Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …