Concepedia

Drugging RNAi

Dirk Haussecker, Mark A. Kay

Science · 2015 · 81 citations · 10 references

Open access

Concepts

PharmacyPharmaceutical BiotechnologyPharmacologyDrug DesignDrug DiscoveryMolecular PharmacologyDrug-target Interaction MappingNucleic Acid Drug DesignRna EditingRna EngineeringRna InterferenceRna-binding ProteinsEarly Drug DiscoveryRnai Pathway Regulation

Abstract

RNAi therapeutics are emerging as a major drug discovery engine

References

10

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Widespread siRNA “off-target” transcript silencing mediated by seed region sequence complementarity

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Life Sciences, Genetics, Molecular Biology +17

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

Teresa Coelho, David Adams, Ana Silva et al. · New England Journal of Medicine · 2013 · 961 citations · Full text

Life Sciences, Molecular Biology, Molecular Medicine +15

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary<i>N</i>-acetyl galactosamine improves potency 10-fold in mice

Thazha P. Prakash, Mark J. Graham, Jinghua Yu et al. · Nucleic Acids Research · 2014 · 597 citations · Full text

Pharmacy, Molecular Pharmaceutics, Pharmacology +17

The Loop Position of shRNAs and Pre-miRNAs Is Critical for the Accuracy of Dicer Processing In Vivo

Shuo Gu, Lan Jin, Yue Zhang et al. · Cell · 2012 · 293 citations · Full text

Life Sciences, Genetics, Molecular Biology +15