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

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 sequ...

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Detalles Bibliográficos
Autores principales: de Fougerolles, Antonin, Vornlocher, Hans-Peter, Maraganore, John, Lieberman, Judy
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2007
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7098199/
https://www.ncbi.nlm.nih.gov/pubmed/17541417
http://dx.doi.org/10.1038/nrd2310
Descripción
Sumario: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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