Review paper
The current state and future directions of RNAi-based therapeutics
Abstract
The RNA interference (RNAi) pathway regulates mRNA stability and translation in nearly all human cells. Small double-stranded RNA molecules can efficiently trigger RNAi silencing of specific genes, but their therapeutic use has faced numerous challenges involving safety and potency. However, August 2018 marked a new era for the field, with the US Food and Drug Administration approving patisiran, the first RNAi-based drug. In this Review, we...
Paper Details
Title
The current state and future directions of RNAi-based therapeutics
Published Date
Mar 7, 2019
Volume
18
Issue
6
Pages
421 - 446
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