The field of genome engineering has created new possibilities for gene therapy, including
improved animal models of disease, engineered cell therapies, and in vivo gene repair. The …

Duchenne muscular dystrophy is a highly progressive muscle wasting disorder due to
primary abnormalities in one of the largest genes in the human genome, the DMD gene …

Many common human mesenchymal tumors, including gastrointestinal stromal tumor (GIST),
rhabdomyosarcoma (RMS) and leiomyosarcoma (LMS), feature myogenic differentiation …

For many neuromuscular disorders, including Duchenne muscular dystrophy, spinal
muscular atrophy and myotonic dystrophy, the genetic causes are well known. Gene therapy …

Mutations of the dystrophin DMD gene, essentially deletions of one or several exons, are the
cause of two devastating and to date incurable diseases, Duchenne (DMD) and Becker …

Sequencing of the human genome and numerous advances in molecular techniques have
launched the era of genetic medicine. Increasingly precise technologies for genetic …

Muscular dystrophies are genetic disorders characterized by skeletal muscle wasting and
weakness. Although there is no effective therapy, a number of experimental strategies have …

The past 18 months have seen a strong increase in the number of exciting reports on novel
therapeutic agents for DMD. Exon-skipping agents have been fine-tuned to improve tissue …

The largest human gene is represented by the X-chromosomal dystrophin gene of 2.4
million bases, which encodes for the membrane cytoskeletal protein dystrophin. The …

Duchenne muscular dystrophy is an inherited, progressive muscle-wasting disorder caused
by mutations in the dystrophin gene. An increasing variety of approaches are moving …