G﻿ene-silencing technology gets first drug approval after 20-year wait

Ledford, Heidi

doi:10.1038/d41586-018-05867-7

Cited by 131RNA Interference and Gene DeliveryVirus-based gene therapy researchPharmaceutical Economics and Policy

Gene-silencing technology gets first drug approval after 20-year wait

Open access

Ledford, Heidi·2018·Nature

DOI 10.1038/d41586-018-05867-7

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Abstract

The US Food and Drug Administration’s decision breathes new life into RNA-interference therapies. RNA-interference therapies were once left for dead but now the US Food and Drug Administration has approved patisiran.

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Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
Fire, A., Xu, S., Montgomery, M. K., Kostas, S. A., Driver, S. E., & Mello, C. C. (1998) · Nature · doi:10.1038/35888 · Cited by 15,429 · see citing works
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
Adams, D., Gonzalez-Duarte, A., O’Riordan, W. D., Yang, C.-C., Ueda, M., Kristen, A. V., Tournev, I., Schmidt, H. H., Coelho, T., Berk, J. L., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1716153 · Cited by 3,047 · see citing works

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Authors: Ledford, H.
Corresponding author: Ledford, Heidi
Venue: Nature
Publisher: Springer Science and Business Media LLC
Published: 2018-08-10
Volume: 560
Issue: 7718
Pages: 291-292
ISSN: 0028-0836 (ISSN-L)
1476-4687
DOI: 10.1038/d41586-018-05867-7
OpenAlex: W2885305111
Cited by: 131
References: 2
Citations by year: 2026: 2 · 2025: 8 · 2024: 12 · 2023: 10 · 2022: 20 · 2021: 21 · 2020: 27 · 2019: 26 · 2018: 5
Type: other
Language: en
Open access: hybrid
doi.org
Licence: other-oa
www.springernature.com/gp/researchers/text-and-data-mining (tdm)
www.springernature.com/gp/researchers/text-and-data-mining (vor)
Locations: Nature (publishedVersion, other-oa)
PubMed (publishedVersion)
RePEc: Research Papers in Economics (submittedVersion)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
Keywords: RNA interference, gene silencing, patisiran, FDA drug approval, siRNA therapeutics, drug development
MeSH terms: RNAi Therapeutics, Humans, Time Factors, United States, United States Food and Drug Administration, Drug Approval, Gene Silencing, Amyloid Neuropathies, Familial, RNA, Small Interfering

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