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Nat Rev Drug Discov. 2007 Jun;6(6):443-53.

Interfering with disease: a progress report on siRNA-based therapeutics.

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Alnylam Pharmaceuticals Inc., 300 Third Street, Cambridge, Massachusetts 02142, USA.


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.

[Indexed for MEDLINE]

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