RNA interference (RNAi) is a regulatory mechanism of most eukaryotic cells that uses small double stranded RNA (dsRNA) molecules to direct homology-dependent control of gene activity [1].

Oct 6, 2022 · RNA Editing in our context refers to the directed change from Adenine (A) to Inosine (I) in an RNA molecule with ‘I’ being read as a ‘G’ by the molecular machineries inside a cell.

Apr 3, 2024 · In this Review, we discuss principal considerations and future directions in the design of therapeutic siRNAs, with a particular emphasis on chemistry, the application of informatics, delivery...

Nov 21, 2022 · RNA interference (RNAi) is a fundamental gene-silencing pathway in eukaryotic cells that involves an enzyme called dicer cleaving long pieces of double-stranded RNA into shorter fragments called siRNAs that can cleave complementary mRNA sequences with the help of the RISC complex and argonaute.

May 9, 2024 · RNA interference (RNAi) therapies represent a revolutionary paradigm in medicine, offering innovative ways to manipulate gene expression for therapeutic purposes. The ability to selectively silence or downregulate specific genes has …

RNA interference (RNAi) is a collection of small RNA directed mechanisms that result in sequence specific inhibition of gene expression. The notion that RNAi could lead to a new class of therapeutics caught the attention of many investigators soon after its discovery.

Mar 14, 2025 · Similar to synthetic peptide rusfertide, the RNaseH antisense sapablursen aims to increase hepcidin pathway activity by facilitating its release from the liver. This can be achieved by knocking down its negative regulator TMPRSS6.

Feb 26, 2024 · Two decades of research on RNA interference (RNAi) have transformed a breakthrough discovery in biology into a robust platform for a new class of medicines that modulate mRNA expression.

Jun 24, 2024 · That original RNAi therapy (patisiran) was directed towards neuropathy, but Alnylam has been hoping that such treatments would show benefit for cardiovascular hATTR. To that end, they've been testing their latest model therapy (vutisiran) in cardiomyopathy patients.

5 days ago · RNA interference (RNAi) therapeutics can silence disease-causing gene transcripts, but extrahepatic delivery has been challenging.