Discovery of the first molecular mechanism of action for a small molecule that improves RNA splicing in cells could lead to new therapeutics for the genetic disorder spinal muscular atrophy (SMA) and ...

It’s understood that degenerative diseases like Alzheimers and cancer can make a person’s “golden years” look pretty bleak. However, there’s some hope on the horizon thanks to some new research from ...

It's understood that degenerative diseases like Alzheimers and cancer can make a person's “golden years” look pretty bleak. However, there’s some hope on the horizon thanks to some new research from ...

Scientific insights often reflect a shift in perspective. For Envisagenics and its partners, insights may be gained through a more encompassing view of gene expression errors. This view includes ...

Splicing of SMN2 generally skips exon 7, resulting in the production of a truncated and inactivated form of the SMN protein. NVS-SM1, a new compound reported by Palacino and colleagues, facilitates ...

PITTSBURGH, Pa., Dec. 16, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class ...

A compound already used to treat pneumonia could become a new therapy for an inherited muscular wasting disease, according to researchers at the University of Oregon and the University of Rochester ...

Image: Structure of the antitumor compound spliceostatin A (purple and orange) bound to a spliceosome complex (light blue and pink). Credit: Professor Vlad Pena. A team of scientists has created a ...