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

By A. D. de Fougerolles, H. Vornlocher, J. Maraganore, J. Lieberman. Nature reviews. Drug discovery, 2007

1,247 citations recorded by the source corpus.

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.

Subfields: Cancer Research, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/dd6bcaf7f4e80bba8d3c1ee7611030edd10e1923

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Citations in the corpus

Subcellular targeting strategies for drug design and delivery (2010, background): “Such prodrugs could be used to evade lysosomal degradation and to increase the efficiency of cytosolic release of molecules such as siRNA”
Twenty years of cell-penetrating peptides: from molecular mechanisms to therapeutics (2009, positive): “Several viral and non-viral strategies have been proposed to improve the delivery of either siRNAexpressing vectors or synthetic siRNAs both in cultured cells and in vivo (De Fougerolles et al., 2007; Juliano et al., 2008).”
siRNA delivery systems for cancer treatment. (2009, background): “Anothermerit of siRNA as a therapeutic drug is that awide range of target proteins can be utilized for gene silencing to treat diseases [13].”
A combinatorial library of lipid-like materials for delivery of RNAi therapeutics (2008, background): “dtd" [ ]> The specific reduction, or silencing, of gene expression through RNAi has considerable potential to create a new class of therapeutics that addresses previously untreatable disease”
Nanoparticle-based combination therapy toward overcoming drug resistance in cancer. (2012, background): “The advancement in siRNA technology and its application to cancer treatment promise highly specific therapeutic options that silence target genes [56].”
Discovery and Development of the G-rich Oligonucleotide AS1411 as a Novel Treatment for Cancer (2009, background): “More recently, the phenomenon of RNA interference has garnered much attention and there is considerable excitement about the therapeutic potential of modified double-stranded RNA oligonucleotides (small interfering RNAs or siRNAs) that can specifically knockdown the expression of diseaseassociated g”
Therapeutic delivery of miR-26a inhibits cancer cell proliferation and induces tumor-specific apoptosis (2009, background): “Since the initial discovery of a functional RNA interference (RNAi) system in mammals, significant effort has been devoted to the development of therapeutics that utilize this pathway (de Fougerolles et al., 2007).”