Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

The clustered regularly interspaced short palindromic repeats (CRISPR) renaissance was
catalysed by the discovery that RNA-guided prokaryotic CRISPR-associated (Cas) proteins …

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 …

At present, the idea of genome modification has revolutionized the modern therapeutic
research era. Genome modification studies have traveled a long way from gene …

Gene therapy has long held promise to correct a variety of human diseases and defects.
Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the …

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

The CRISPR-Cas9 RNA-guided DNA endonuclease has contributed to an explosion of
advances in the life sciences that have grown from the ability to edit genomes within living …

Programmable DNA cleavage using CRISPR–Cas9 enables efficient, site-specific genome
engineering in single cells and whole organisms. In the research arena, versatile CRISPR …

Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

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 …