RNAi Therapeutics: Principles, Prospects and Challenges
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 …
RNAi Therapeutics | How RNA Interference 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. The …
RNA interference in the era of nucleic acid therapeutics
2024年2月26日 · 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 drug design: considerations and future directions
2024年4月3日 · 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 …
The current state and future directions of RNAi-based therapeutics
2019年3月7日 · 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 …
Taking RNAi from interesting science to impactful new treatments
2024年5月13日 · Alnylam Pharmaceuticals is translating the promise of RNA interference (RNAi) research into a new class of powerful, gene-based therapies. In this rendering, the green …
25 years of maturation: A systematic review of RNAi in the clinic
2023年9月12日 · This systematic review investigates how the maturation of RNAi therapeutics and developments in delivery platforms, administration routes, and potential targets shape the …
RNAi-Based Therapeutics and Novel RNA Bioengineering …
2023年1月1日 · We overview the pharmacology of the four FDA-approved siRNA medications (patisiran, givosiran, lumasiran, and inclisiran) as well as five siRNAs and several miRNA …
RNAi therapeutics: a potential new class of pharmaceutical drugs
Therapeutics based on RNA interference (RNAi) offer a powerful method for rapidly identifying specific and potent inhibitors of disease targets from all molecular classes. Numerous proof-of …
The Growing Class of Novel RNAi Therapeutics - PMC
Herein, we discuss the RNAi mechanism of action and current criteria for distinguishing between miRNAs and siRNAs while summarizing the common and unique chemistry and molecular …
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