The colorful threads in this image are muscle fibers. This spotted rainbow illustrates a potential gene therapy approach to treating Duchenne muscular dystrophy (DMD) – a rare, debilitating, and fatal ...

DMD is a type of muscular dystrophy ... People with DMD have mutations (changes) in the gene that cause a loss of dystrophin protein in cells. The lack of dystrophin protein causes muscle cells ...

It is debatable, however, whether this creates a problem for society, because even though the frequency of the lethal mutations would increase, the number of babies born with DMD would decrease.

Duchenne muscular dystrophy is a genetic condition that leads to progressive muscle weakness and degeneration. It’s caused by mutations in the DMD gene, which carries the instructions for making ...

Using CRISPR-Cpf1 gene editing, researchers have fixed mutations that cause a form of muscular dystrophy in cultured human cardiomyocytes and a mouse model. “The authors use CRISPR-Cfp1 to correct the ...

Duchenne muscular dystrophy causes progressive muscle degeneration and weakness, but physical exercise should still be part of a comprehensive treatment strategy. Duchenne muscular dystrophy (DMD ...

NS Pharma’s Viltepso (viltolarsen) has been approved in patients who have confirmed mutation of the DMD gene that is amenable to exon 53 skipping. The DMD gene is made up of 79 exons ...

The funding will be used to continue the clinical development of lead oligonucleotide PGN-EDO51 for DMD, as well as two other oligo drugs PGN-EDODM1 for myotonic dystrophy type 1 (DM1 ...

Here, the authors report compact type V-K CAST discovered from genome ... is a fatal genetic disorder caused by mutations in the DMD gene. Here, the authors develop MyoAAV-UA, a compact utrophin ...

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...