RNAi Therapeutics: Principles, Prospects and Challenges - PMC
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 …
Pioneering RNAi Therapeutics - How RNAi Works | Alnylam® …
RNAi therapeutics is an innovative new class of medicines based on RNA interference (RNAi)—a breakthrough discovery in understanding how genes are regulated naturally within cells.
RNAi-based therapeutics–current status, challenges and prospects
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 …
The current state and future directions of RNAi-based therapeutics
Mar 7, 2019 · In this Review, we discuss key advances in the design and development of RNAi drugs leading up to this landmark achievement, the state of the current clinical pipeline and …
RNAi-based drug design: considerations and future directions
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 …
RNA interference in the era of nucleic acid therapeutics
Feb 26, 2024 · Here we provide an overview of the trajectory of small-interfering RNA (siRNA) drug development, including the first approval in 2018 of a liver-targeted siRNA interference …
RNAi-Based Therapeutics and Novel RNA Bioengineering …
Mar 14, 2022 · We overview the pharmacology of the four FDA-approved siRNA medications (patisiran, givosiran, lumasiran, and inclisiran) as well as five siRNAs and several miRNA …
Atalanta Therapeutics Publishes Preclinical Data Supporting ...
1 day ago · Atalanta Therapeutics, a biotechnology company pioneering RNA interference (RNAi) for the treatment of neurological diseases, today announced the publication of preclinical data …
Alnylam Announces FDA Approval of Amvuttra (vutrisiran), the First RNAi …
Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger …
RNAi Therapies: Drugging the Undruggable - Science
Jun 11, 2014 · RNA interference (RNAi) therapy is a rapidly emerging platform for personalized cancer treatment. Recent advances in small interfering RNA delivery and target selection …