Researchers from Kyoto University demonstrate how a dual CRISPR RNA method restored dystrophin protein function in induced pluripotent stem cells derived from Duchenne muscular dystrophy (DMD) ...

Duchenne muscular dystrophy (DMD) is a severe and progressive muscle disorder in which muscle degeneration and necrosis occur due to the absence of a protein called dystrophin. There is no cure for ...

Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness and wasting. It is an X-linked recessive disorder that occurs due to changes in the DMD gene, which ...

Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...

A new study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its ...

"By understanding how these proteins function differently in various tissues, we're one step closer to designing treatments that target the root causes of DMD." The study reveals that dystrophin's CT ...

New data demonstrates the ability of UMD technology to deliver genetic medicines, allowing for full-length human dystrophin protein expression in skeletal, heart and diaphragm muscle tissue in murine ...

Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new ...

Gene therapy may have the potential to cure Duchenne muscular dystrophy, but early results from clinical trials have not been as promising as researchers hoped. Research is ongoing. Duchenne muscular ...