PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and ...

Muscular dystrophy is caused by a lack of certain proteins that are needed for muscle structure and function. Specifically, many of the types of MD are caused by a lack of a protein called dystrophin, ...

The treatment led to a substantial reduction in DMPK RNA along with splicing defect corrections, as measured by the composite alternative ... DYNE-251, designed with the same platform as DYNE-101, is ...

WVE-N531 is an exon skipping oligonucleotide designed to induce production of endogenous, functional dystrophin protein for the treatment of boys with DMD amenable to exon 53 skipping. Key ...

WVE-N531 is an exon skipping oligonucleotide designed to induce production of endogenous, functional dystrophin protein for the treatment of boys with DMD amenable to exon 53 skipping. Key ...

The trial data demonstrated a reduction in DMPK RNA levels, the molecular target of DYNE-101, and robust splicing correction associated with improvements in multiple ... for Duchenne muscular ...

Dyne measured splicing in all study participants using ... Dyne previously reported that DYNE-251 demonstrated unprecedented dystrophin expression and functional improvement on multiple measures ...

The work will be of broad interest to cell biologists and cardiac electrophysiologists. Therefore, in this study we explored whether lncDACH1 regulates Nav1.5 by interacting with dystrophin. We found ...

Peer ReviewDownload a summary of the editorial decision process including editorial decision letters, reviewer comments and author responses to feedback. XPO7 haploinsufficiency increases the risk of ...