Blocking sense-strand activity improves potency, safety and specificity of anti-hepatitis B virus short hairpin RNA.

PubWeight™: 0.80‹?›

🔗 View Article (PMID 27473329)

Published in EMBO Mol Med on September 01, 2016

Authors

Thomas Michler1, Stefanie Große2, Stefan Mockenhaupt2, Natalie Röder3, Ferdinand Stückler4, Bettina Knapp4, Chunkyu Ko3, Mathias Heikenwalder3, Ulrike Protzer5, Dirk Grimm6

Author Affiliations

1: Institute of Virology, Technische Universität München/Helmholtz Zentrum München, München, Germany German Center for Infection Research (DZIF), partner site München, München, Germany.
2: Department of Infectious Diseases/Virology, Cluster of Excellence CellNetworks, Heidelberg University Hospital, Heidelberg, Germany BioQuant, University of Heidelberg, Heidelberg, Germany.
3: Institute of Virology, Technische Universität München/Helmholtz Zentrum München, München, Germany.
4: Institute of Computational Biology, Helmholtz Zentrum München, Neuherberg, Germany.
5: Institute of Virology, Technische Universität München/Helmholtz Zentrum München, München, Germany German Center for Infection Research (DZIF), partner site München, München, Germany protzer@tum.de protzer@helmholtz-muenchen.de dirk.grimm@bioquant.uni-heidelberg.de.
6: Department of Infectious Diseases/Virology, Cluster of Excellence CellNetworks, Heidelberg University Hospital, Heidelberg, Germany BioQuant, University of Heidelberg, Heidelberg, Germany protzer@tum.de protzer@helmholtz-muenchen.de dirk.grimm@bioquant.uni-heidelberg.de.

Articles cited by this

MEGA6: Molecular Evolutionary Genetics Analysis version 6.0. Mol Biol Evol (2013) 112.81

A mammalian microRNA expression atlas based on small RNA library sequencing. Cell (2007) 34.03

Functional siRNAs and miRNAs exhibit strand bias. Cell (2003) 19.15

Expression profiling reveals off-target gene regulation by RNAi. Nat Biotechnol (2003) 16.63

Rational siRNA design for RNA interference. Nat Biotechnol (2004) 14.16

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med (2006) 13.51

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med (2011) 11.01

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature (2006) 10.95

High-level hepatitis B virus replication in transgenic mice. J Virol (1995) 5.81

RNA interference in adult mice. Nature (2002) 5.33

Sodium taurocholate cotransporting polypeptide is a functional receptor for human hepatitis B and D virus. Elife (2012) 5.18

Short interfering RNAs can induce unexpected and divergent changes in the levels of untargeted proteins in mammalian cells. Proc Natl Acad Sci U S A (2004) 4.85

A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J Virol (1987) 4.57

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci U S A (2008) 4.53

Estimations of worldwide prevalence of chronic hepatitis B virus infection: a systematic review of data published between 1965 and 2013. Lancet (2015) 4.37

A framework for oligonucleotide microarray preprocessing. Bioinformatics (2010) 3.97

In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J Virol (2008) 3.79

Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs. Nat Biotechnol (2009) 3.66

Target mRNA abundance dilutes microRNA and siRNA activity. Mol Syst Biol (2010) 3.37

Inhibition of hepatitis B virus in mice by RNA interference. Nat Biotechnol (2003) 3.15

Self-complementary AAV vectors; advances and applications. Mol Ther (2008) 3.10

Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med (2014) 2.92

Off-target effects by siRNA can induce toxic phenotype. RNA (2006) 2.89

Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo. Mol Ther (2008) 2.66

Vectors expressing efficient RNA decoys achieve the long-term suppression of specific microRNA activity in mammalian cells. Nucleic Acids Res (2009) 2.52

DAVID-WS: a stateful web service to facilitate gene/protein list analysis. Bioinformatics (2012) 2.14

Living in the liver: hepatic infections. Nat Rev Immunol (2012) 2.03

Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic. Mol Ther (2008) 2.01

Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. J Clin Invest (2010) 1.91

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas. Nat Genet (2015) 1.86

Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose. Mol Ther (2007) 1.85

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature (2013) 1.80

Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs. RNA (2008) 1.70

Use of RNA polymerase II to transcribe artificial microRNAs. Methods Enzymol (2005) 1.68

The N domain of Argonaute drives duplex unwinding during RISC assembly. Nat Struct Mol Biol (2012) 1.55

Coexpression of Argonaute-2 enhances RNA interference toward perfect match binding sites. Proc Natl Acad Sci U S A (2008) 1.49

Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma? Hum Gene Ther (2015) 1.25

Production methods for gene transfer vectors based on adeno-associated virus serotypes. Methods (2002) 1.23

Robust RNAi enhancement via human Argonaute-2 overexpression from plasmids, viral vectors and cell lines. Nucleic Acids Res (2013) 1.20

Expressed anti-HBV primary microRNA shuttles inhibit viral replication efficiently in vitro and in vivo. Mol Ther (2008) 1.15

Induction of stable RNA interference in mammalian cells. Gene Ther (2006) 1.15

Mechanisms of HBV-induced hepatocellular carcinoma. J Hepatol (2016) 1.13

RDAVIDWebService: a versatile R interface to DAVID. Bioinformatics (2013) 1.10

Rational design leads to more potent RNA interference against hepatitis B virus: factors effecting silencing efficiency. Mol Ther (2008) 1.10

Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice. Mol Ther (2009) 1.09

Knockdown of TNFR1 by the sense strand of an ICAM-1 siRNA: dissection of an off-target effect. Nucleic Acids Res (2007) 1.06

Molecular dissection of human Argonaute proteins by DNA shuffling. Nat Struct Mol Biol (2013) 1.03

Comparative study of anti-hepatitis B virus RNA interference by double-stranded adeno-associated virus serotypes 7, 8, and 9. Mol Ther (2008) 1.02

The dose can make the poison: lessons learned from adverse in vivo toxicities caused by RNAi overexpression. Silence (2011) 1.00

Long-term inhibition of hepatitis B virus in transgenic mice by double-stranded adeno-associated virus 8-delivered short hairpin RNA. Gene Ther (2006) 1.00

Efficient silencing of gene expression with modular trimeric Pol II expression cassettes comprising microRNA shuttles. Nucleic Acids Res (2009) 0.98

Adeno-associated Vector Toxicity-To Be or Not to Be? Mol Ther (2015) 0.98

HBVRegDB: annotation, comparison, detection and visualization of regulatory elements in hepatitis B virus sequences. Virol J (2007) 0.97

Modeling RNA interference in mammalian cells. BMC Syst Biol (2011) 0.96

Effective inhibition of HBV replication in vivo by anti-HBx short hairpin RNAs. Mol Ther (2005) 0.95

5' Triphosphorylated small interfering RNAs control replication of hepatitis B virus and induce an interferon response in human liver cells and mice. Gastroenterology (2011) 0.92

Both strands of siRNA have potential to guide posttranscriptional gene silencing in mammalian cells. PLoS One (2009) 0.91

Diagnostic and therapeutic progress of multi-drug resistance with anti-HBV nucleos(t)ide analogues. World J Gastroenterol (2012) 0.90

Direct effects of hepatitis B virus-encoded proteins and chronic infection in liver cancer development. Dig Dis (2013) 0.90

E Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal-Tailored Acceleration of AAV Evolution. Mol Ther (2015) 0.90

Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model. Mol Ther (2012) 0.89

Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria. J Hepatol (2016) 0.88

Safety profile of recombinant adeno-associated viral vectors: focus on alipogene tiparvovec (Glybera®). Expert Rev Clin Pharmacol (2013) 0.86

Adeno-associated viral vectors for the treatment of hemophilia. Hum Mol Genet (2015) 0.85

Weak base pairing in both seed and 3' regions reduces RNAi off-targets and enhances si/shRNA designs. Nucleic Acids Res (2014) 0.84

Alleviation of off-target effects from vector-encoded shRNAs via codelivered RNA decoys. Proc Natl Acad Sci U S A (2015) 0.84

Hepatitis B virus infection enhances susceptibility toward adeno-associated viral vector transduction in vitro and in vivo. Hepatology (2014) 0.83

Managing microRNAs with vector-encoded decoy-type inhibitors. Mol Ther (2013) 0.82

Mechanistic insights on the Dicer-independent AGO2-mediated processing of AgoshRNAs. RNA Biol (2015) 0.81

Strand antagonism in RNAi: an explanation of differences in potency between intracellularly expressed siRNA and shRNA. Nucleic Acids Res (2011) 0.81

Estimating the global prevalence of hepatitis B. Lancet (2015) 0.80

Asymmetry in siRNA design. Gene Ther (2009) 0.80

Therapeutic expression of hairpins targeting apolipoprotein B100 induces phenotypic and transcriptome changes in murine liver. Gene Ther (2013) 0.80