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 ...

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 ...

A compound already used to treat pneumonia could become a new therapy for an inherited muscular wasting disease. Researchers report that pentamidine might be adapted to counter genetic splicing ...

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 ...

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 ...

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 ...

Figure 1: The splicing processing defect in ATM intron 20 does not affect canonical splicing regulatory elements. Figure 2: Base-pairing between ISPE and U1 RNA regulates intron splicing processing.