Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre ...

Dystrophin, along with members of the dystrophin-associated protein complex (DAPC), link the intracellular cytoskeleton with the extracellular matrix and protect the muscle cell from damage caused ...

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 ...

Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production. Studies of the new protein ...

Furthermore, the researchers found for the first time a shorter form of dystrophin (DP71), which is synthesized as a protein in the spleen. "This dystrophin variant is apparently not affected by ...

Dystrophin protein recovery did not match with the ddPCR results. The dual CRISPR-Cas3 system could induce MES in DMD iPSCs with several mutations and restore dystrophin, indicating the wide ...