Disclaimer: AlphaGalileo is not responsible for the accuracy of content posted to AlphaGalileo by contributing institutions or for the use of any information through the AlphaGalileo system.
PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and ...
RNA interference (RNAi) therapeutics are based on a natural process by which RNA sequences ... For people with Duchenne muscular dystrophy, these drugs have been a significant step towards their care.
This abnormal RNA accumulates and disturbs the proper production of proteins important for muscle ... for patients with disease-causing mutations in the DMD gene amenable to exon 51 skipping. Dyne ...
Researchers have now uncovered the 3D structure of the RNA enzyme SAMURI. Their study provides insights into the development of ribozymes and the evolution of catalytically active RNA. Researchers ...
Exon skipping, which ultimately leads to functional ... This was the first-ever proof of RNA editing in humans, according to the company. Data showed that 006 was successful in ...