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https://phys.org/news/2018-08-rna-drug.html
Aug 17, 2018 · The Food and Drug Administration recently approved the very first therapeutic small interfering RNA (siRNA), Onpattro (patisiran), to treat nerve damage caused by a rare disease called hereditary transthyretin-mediated amyloidosis (hATTR). Hereditary transthyretin-mediated amyloidosis affects about 50,000 people worldwide.Author: Thomas Schmittgen
https://www.alnylam.com/our-science/the-science-of-rnai/
RNA interference (RNAi) is a breakthrough in understanding how genes are regulated in cells. It also represents a completely new approach to drug discovery and development. The 2006 Nobel Prize for Physiology or Medicine, awarded to Craig Mello and Andrew Fire, recognized the importance of RNAi as a major scientific discovery.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3378126/
Mar 16, 2009 · 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. ... Targeted delivery is a major issue in many other more developed drug ...Author: Katrin Tiemann, John J. Rossi
https://www.nature.com/articles/s41573-019-0017-4
Mar 07, 2019 · 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.Author: Ryan L. Setten, Ryan L. Setten, John J. Rossi, John J. Rossi, Si-ping Han, Si-ping Han
https://www.nature.com/articles/d41586-018-05867-7
Aug 10, 2018 · US regulators have approved the first therapy based on RNA interference (RNAi), a technique that can be used to silence specific genes linked to disease. The drug, patisiran, targets a rare condition that can impair heart and nerve function. The approval, announced by the US Food and Drug Administration on 10 August,...Author: Heidi Ledford
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