Alternative RNA splicing is like a movie editor cutting and rearranging scenes from the same footage to create different versions of a film.

Japan-based Ono Pharmaceuticals has secured the global rights to sapablursen, an RNA-targeting therapy developed by Ionis ...

Avidity Biosciences, Inc. (NASDAQ:RNA) has emerged as a frontrunner in ... Del-zota: Designed for DMD exon 44 skipping, this therapy has generated considerable excitement and could potentially ...

Researchers at Case Western Reserve University have discovered molecules that present the potential to drive the development ...

Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) released topline results from Part 2 of a Phase 1/2 study of ARO-C3, the ...

Researchers found that a protein from tardigrades could help astronauts ward off the damage from space radiation.

A vast search of natural diversity has led scientists at MIT’s McGovern Institute and the Broad Institute of MIT and Harvard ...

A new review article highlights the transformative role of circular RNA (circRNA) in cancer, revealing its potential as both a key player in tumor biology and a promising avenue for future therapies.

Cells have a natural editing system that allows them to rearrange genetic instructions to create different proteins from the ...

an RNA-based therapeutic targeting metastatic cancers. The decision follows a favorable safety review of the third cohort, allowing the planned dose increase by approximately 50 percent.

The one-time therapy appears to correct the mutation that causes the disease, known as Alpha-1 antitrypsin deficiency (AATD), ...