Series A financing co-led by Balyasny Asset Management, venBio, and Janus Henderson InvestorsRugonersen, a potentially best-in-class antisense ...
When the drug fomivirsen was approved by the FDA in 1998 for the treatment of cytomegalovirus retinitis in patients with HIV/AIDs, it was hailed as a milestone in drug discovery because it was the ...
The function of non-coding RNA in the cell has long been a mystery to researchers. Unlike coding RNA, non-coding RNA does not produce proteins – yet it exists in large quantities. A research team from ...
An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome. This breakthrough was possible because of the novel findings, also presented ...
Scientists at St. Jude Children's Research Hospital have found that they can reverse the effects of HNRNPH2-related neurodevelopmental disorder using antisense oligonucleotides (ASOs) in preclinical ...
Gene silencing by RNA interference (RNAi) has become a powerful tool for functional genomics. In the January 25 Science, Tijsterman et al. report a gene-silencing method induced by short (25 ...
Courtesy of Antisense Therapeutics Ltd. Antisense strategies use polymeric nucleic acids or nucleic acid analogs to bind to and silence specific messenger RNAs. The silencing can be caused either by ...
An antisense therapy developed by Joel D. Richter, Ph.D., Sneha Shah, Ph.D., and Jonathan K. Watts, Ph.D., at UMass Chan Medical School and Elizabeth Berry-Kravis, MD ...
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