PepGen shifts focus to PGN-EDODM1 for Myotonic Dystrophy Type 1 after discontinuing PGN-EDO51 for Duchenne Muscular Dystrophy ...

Splicing of pre-messenger RNA is regulated differently in the brain compared with other tissues. Recognition of aberrations in splicing events that are associated with neurological disease has ...

This article was review by Thomas Cooper, MD from Baylor College of Medicine. Stay up to date on the latest science with Brush Up Summaries. Located throughout the pre-mRNA are short, cis-acting ...

Our cells have to generate a massive number of proteins, and there is a carefully orchestrated procedure for doing so. It starts when active genes are transcribed into RNA, and while that RNA ...

Splicing factor mutations are particularly prevalent in MDS, a group of heterogeneous hematological disorders characterized by defective blood stem cells and a high risk of leukemia development.

A collaborative study published today in the journal Cell Reports provides evidence for a new molecular cause for neurodegeneration in Alzheimer’s disease. The study, led by researchers at Baylor ...