The promises and pitfalls of RNA-interference-based therapeutics

By D. Castanotto, John J. Rossi. Nature, 2009

1,205 citations recorded by the source corpus.

The discovery that gene expression can be controlled by the Watson–Crick base-pairing of small RNAs with messenger RNAs containing complementary sequence — a process known as RNA interference — has markedly advanced our understanding of eukaryotic gene regulation and function. The ability of short RNA sequences to modulate gene expression has provided a powerful tool with which to study gene function and is set to revolutionize the treatment of disease. Remarkably, despite being just one decade from its discovery, the phenomenon is already being used therapeutically in human clinical trials, and biotechnology companies that focus on RNA-interference-based therapeutics are already publicly traded.

Subfields: Cardiology and Cardiovascular Medicine, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/b18a34e95d2dae3a7b1b6f4e0202a2e7d1e95561

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Citations in the corpus

Delivery of siRNA Therapeutics: Barriers and Carriers (2010, background): “... complexing siRNA with a positively charged vector (e.g., cationic cell penetrating peptides, cationic polymers and dendrimers, and cationic lipids); conjugating siRNA with small molecules (e.g., cholesterol, bile acids, and lipids), polymers, antibodies, and RNAs; and encapsulating siRNA in nano”
Mammalian cell transfection: the present and the future (2010, background): “Despite the wide use of siRNA, large efforts are still being made to develop more effective, safe, and reliable methods to deliver siRNAs into cells, because of the great potential of RNAi in clinical use to treat diseases [ 33 ].”
Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles (2010, background): “1a) containing: (1) a linear, cyclodextrin-based polymer (CDP), (2) a human transferrin protein (TF) targeting ligand displayed on the exterior of the nanoparticle to engage TF receptors (TFR) on the surface of the cancer cells, (3) a hydrophilic polymer (polyethylene glycol (PEG) used to promote na”
Principles of early drug discovery (2011, methodology): “After integration into the RISC, siRNAs basepair to their target mRNA and induce cleavage of the mRNA, thereby preventing it from being used as a translation template (reviewed in Castanotto and Rossi, 2009).”
Multi-task learning for cross-platform siRNA efficacy prediction: an in-silico study (2010, background): “Nowadays, RNAi has become an effective tool to inhibit gene expression, serving as a potential therapeutic strategy in viral diseases, drug target discovery and cancer therapy [ 2 ].”
RNA-based Therapeutics- Current Progress and Future Prospects (2012, background): “…of reasons, such as ability of siRNAs to elicit potent targetspecific knockdown of any mRNA, ease of siRNA design and screening against the mRNA target, and achievement of longlasting silencing as the siRNA can retain its catalytic activity in RISC for long periods (Castanotto and Rossi, 2009).”