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 ...

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 ...

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 ...

Eight Northwestern researchers published a study April 29 detailing how RNA therapy could be used before birth to combat a ...

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 ...

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 ...

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 ...

Various sayings equate going fast with going alone. But none of them apply to the gene expression journey described by a research team from the University of Göttingen. The team, led by Heike Krebber, ...

A single injection into the bloodstream of a mouse corrected a mutation linked to autism in neurons throughout the brain and ...

An antisense therapy developed by Joel D. Richter, PhD, Sneha Shah, PhD, and Jonathan K. Watts, PhD, at UMass Chan Medical School and Elizabeth Berry-Kravis, MD, PhD, at RUSH University Medical Center ...