Interfering with disease: a progress report on siRNA-based therapeutics
Identifieur interne : 001664 ( Main/Exploration ); précédent : 001663; suivant : 001665Interfering with disease: a progress report on siRNA-based therapeutics
Auteurs : Antonin De Fougerolles [États-Unis] ; Hans-Peter Vornlocher [États-Unis, Allemagne] ; John Maraganore [États-Unis] ; Judy Lieberman [États-Unis]Source :
- Nature Reviews Drug Discovery [ 1474-1776 ] ; 2007-06.
Abstract
RNA interference (RNAi) quietly crept into biological research in the 1990s when unexpected gene-silencing phenomena in plants and flatworms first perplexed scientists. Following the demonstration of RNAi in mammalian cells in 2001, it was quickly realized that this highly specific mechanism of sequence-specific gene silencing might be harnessed to develop a new class of drugs that interfere with disease-causing or disease-promoting genes. Here we discuss the considerations that go into developing RNAi-based therapeutics starting from in vitro lead design and identification, to in vivo pre-clinical drug delivery and testing. We conclude by reviewing the latest clinical experience with RNAi therapeutics.
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DOI: 10.1038/nrd2310
Affiliations:
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xml:lang="fr">Allemagne</country><wicri:regionArea>Hans-Peter Vornlocher is at Alnylam Europe AG, Fritz-Hornschuch-Str. 9, Kulmbach 95326</wicri:regionArea><wicri:noRegion>95326</wicri:noRegion><wicri:noRegion>Kulmbach 95326</wicri:noRegion></affiliation></author><author><name sortKey="Maraganore, John" sort="Maraganore, John" uniqKey="Maraganore J" first="John" last="Maraganore">John Maraganore</name><affiliation wicri:level="1"><country xml:lang="fr" wicri:curation="lc">États-Unis</country><wicri:regionArea>Antonin de Fougerolles and John Maraganore are at Alnylam Pharmaceuticals Inc., 300 Third Street, Cambridge, Massachusetts 02142</wicri:regionArea><wicri:noRegion>Massachusetts 02142</wicri:noRegion></affiliation></author><author><name sortKey="Lieberman, Judy" sort="Lieberman, Judy" uniqKey="Lieberman J" first="Judy" last="Lieberman">Judy Lieberman</name><affiliation wicri:level="1"><country xml:lang="fr" wicri:curation="lc">États-Unis</country><wicri:regionArea>Judy Lieberman is 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xml:lang="eng">RNA interference (RNAi) quietly crept into biological research in the 1990s when unexpected gene-silencing phenomena in plants and flatworms first perplexed scientists. Following the demonstration of RNAi in mammalian cells in 2001, it was quickly realized that this highly specific mechanism of sequence-specific gene silencing might be harnessed to develop a new class of drugs that interfere with disease-causing or disease-promoting genes. Here we discuss the considerations that go into developing RNAi-based therapeutics starting from in vitro lead design and identification, to in vivo pre-clinical drug delivery and testing. 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