Advancements in RNAi Therapeutics and Delivery
Advancements in RNAi Therapeutics and Delivery
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This overview explores RNA interference (RNAi) mechanisms, highlighting how it targets undruggable proteins through Ago2-mediated mRNA cleavage and the roles of siRNA and ASO. It discusses delivery systems like LNP and GalNAc, addressing challenges in endosomal escape and toxicity reduction via 2' modifications. The clinical translation section reviews approved RNAi drugs, such as Zilebesiran and olpasiran, emphasizing their potential for durable gene silencing and the need for advancements...