StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

Researchers have developed a simple model system that can be used to break down fibrils -- the cause of numerous disorders including Alzheimer's and Parkinson's disease -- into their constituent ...

Heart muscle cells grown from stem cells show promise in monkeys with a heart problem that typically results from a heart ...

The origin of many diseases such as Alzheimer's or Parkinson's can be found at the molecular level in our body, in other ...

This important manuscript provides insights into the competition between Splicing Factor 1 (SF1) and Quaking (QKI) for binding at the ACUAA branch point sequence in a model intron, regulating exon ...