A status report on RNAi therapeutics

[1]  K. G. Rajeev,et al.  Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

[2]  K. G. Rajeev,et al.  Rational design of cationic lipids for siRNA delivery , 2010, Nature Biotechnology.

[3]  R. Hickerson,et al.  First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

[4]  Kathryn A. Whitehead,et al.  Lipid-like materials for low-dose, in vivo gene silencing , 2010, Proceedings of the National Academy of Sciences.

[5]  S. Stirdivant,et al.  Evaluation of efficacy, biodistribution, and inflammation for a potent siRNA nanoparticle: effect of dexamethasone co-treatment. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

[6]  P. Linsley,et al.  Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application , 2010, Nature Reviews Drug Discovery.

[7]  Anton P. McCaffrey,et al.  Systemic administration of optimized aptamer-siRNA chimeras promotes regression of PSMA-expressing tumors , 2009, Nature Biotechnology.

[8]  Svetlana Shulga Morskaya,et al.  RNA Interference-Mediated Silencing of the Respiratory Syncytial Virus Nucleocapsid Defines a Potent Antiviral Strategy , 2009, Antimicrobial Agents and Chemotherapy.

[9]  Martin L. Miller,et al.  Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs , 2009, Nature Biotechnology.

[10]  A. Judge,et al.  siRNA and innate immunity. , 2009, Oligonucleotides.

[11]  Michael S. Goldberg,et al.  Development of lipidoid-siRNA formulations for systemic delivery to the liver. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

[12]  M. Czech,et al.  Orally delivered siRNA targeting macrophage Map4k4 suppresses systemic inflammation , 2009, Nature.

[13]  A. Judge,et al.  Confirming the RNAi-mediated mechanism of action of siRNA-based cancer therapeutics in mice. , 2009, The Journal of clinical investigation.

[14]  M. Manoharan,et al.  Direct CNS delivery of siRNA mediates robust silencing in oligodendrocytes. , 2009, Oligonucleotides.

[15]  Daniel G. Anderson,et al.  Knocking down barriers: advances in siRNA delivery , 2009, Nature Reviews Drug Discovery.

[16]  M. Poidinger,et al.  Sequence determinants of innate immune activation by short interfering RNAs , 2009, BMC Immunology.

[17]  B. Wiedenmann,et al.  Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression. , 2008, Cancer research.

[18]  A. Judge,et al.  Misinterpreting the therapeutic effects of small interfering RNA caused by immune stimulation. , 2008, Human gene therapy.

[19]  Robert Langer,et al.  Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates , 2008, Proceedings of the National Academy of Sciences.

[20]  Ruiwen Zhang,et al.  Antisense, RNAi, and gene silencing strategies for therapy: mission possible or impossible? , 2008, Drug discovery today.

[21]  D. Haussecker,et al.  The business of RNAi therapeutics. , 2008, Human gene therapy.

[22]  Justine R. Smith,et al.  Sequence- and target-independent angiogenesis suppression by siRNA via TLR3 , 2008, Nature.

[23]  S. Elbashir,et al.  Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV). , 2008, Antiviral research.

[24]  A. Akinc,et al.  Delivering silence: advancements in developing siRNA therapeutics. , 2008, Current opinion in drug discovery & development.

[25]  D. Peer,et al.  Systemic Leukocyte-Directed siRNA Delivery Revealing Cyclin D1 as an Anti-Inflammatory Target , 2008, Science.

[26]  E. Swayze,et al.  The Medicinal Chemistry of Oligonucleotides , 2008 .

[27]  V. A. Stewart,et al.  Activation of Innate Immunity in Healthy Macaca mulatta Macaques by a Single Subcutaneous Dose of GMP CpG 7909: Safety Data and Interferon-Inducible Protein-10 Kinetics for Humans and Macaques , 2007, Clinical and Vaccine Immunology.

[28]  K. G. Rajeev,et al.  Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits , 2007, Proceedings of the National Academy of Sciences.

[29]  M. Stoffel,et al.  Mechanisms and optimization of in vivo delivery of lipophilic siRNAs , 2007, Nature Biotechnology.

[30]  Wolfgang A. Weber,et al.  Impact of tumor-specific targeting on the biodistribution and efficacy of siRNA nanoparticles measured by multimodality in vivo imaging , 2007, Proceedings of the National Academy of Sciences.

[31]  Darren H. Wakefield,et al.  Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes , 2007, Proceedings of the National Academy of Sciences.

[32]  A. Elmaagacli,et al.  Therapeutic application of small interfering RNA directed against bcr-abl transcripts to a patient with imatinib-resistant chronic myeloid leukaemia , 2007, Clinical and Experimental Medicine.

[33]  A. Vicari,et al.  Safety, Pharmacokinetics and Immune Effects in Normal Volunteers of CPG 10101 (ACTILON™), an Investigational Synthetic Toll-like Receptor 9 Agonist , 2007, Antiviral therapy.

[34]  Judy Lieberman,et al.  Interfering with disease: a progress report on siRNA-based therapeutics , 2007, Nature Reviews Drug Discovery.

[35]  J. Lieberman,et al.  Selective gene silencing in activated leukocytes by targeting siRNAs to the integrin lymphocyte function-associated antigen-1 , 2007, Proceedings of the National Academy of Sciences.

[36]  John G Doench,et al.  Comparison of siRNA-induced off-target RNA and protein effects. , 2007, RNA.

[37]  L. Lim,et al.  Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity. , 2006, RNA.

[38]  Theresa A. Storm,et al.  Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways , 2006, Nature.

[39]  Matthias John,et al.  RNAi-mediated gene silencing in non-human primates , 2006, Nature.

[40]  Anastasia Khvorova,et al.  3′ UTR seed matches, but not overall identity, are associated with RNAi off-targets , 2006, Nature Methods.

[41]  P. Campochiaro,et al.  Suppression of ocular neovascularization with siRNA targeting VEGF receptor 1 , 2006, Gene Therapy.

[42]  J. Lieberman,et al.  An siRNA-based microbicide protects mice from lethal herpes simplex virus 2 infection , 2006, Nature.

[43]  K. Livak,et al.  Real-time quantification of microRNAs by stem–loop RT–PCR , 2005, Nucleic acids research.

[44]  Keith Bowman,et al.  Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs , 2005, Nature Biotechnology.

[45]  D. Stokoe,et al.  Direct comparison of the specificity of gene silencing using antisense oligonucleotides and RNAi. , 2005, The Biochemical journal.

[46]  Judy Lieberman,et al.  Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors , 2005, Nature Biotechnology.

[47]  M. Sioud.  Induction of inflammatory cytokines and interferon responses by double-stranded and single-stranded siRNAs is sequence-dependent and requires endosomal localization. , 2005, Journal of molecular biology.

[48]  A. Judge,et al.  Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA , 2005, Nature Biotechnology.

[49]  S. Akira,et al.  Sequence-specific potent induction of IFN-α by short interfering RNA in plasmacytoid dendritic cells through TLR7 , 2005, Nature Medicine.

[50]  Joel S. Parker,et al.  Transcriptional Profiling of the Left and Median Liver Lobes of Male F344/N Rats Following Exposure to Acetaminophen , 2005, Toxicologic pathology.

[51]  S. Barik,et al.  Inhibition of respiratory viruses by nasally administered siRNA , 2005, Nature Medicine.

[52]  Matthias John,et al.  Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs , 2004, Nature.

[53]  K. Elenitoba-Johnson,et al.  Quantitative Proteomic and Transcriptional Analysis of the Response to the p38 Mitogen-activated Protein Kinase Inhibitor SB203580 in Transformed Follicular Lymphoma Cells*S , 2004, Molecular & Cellular Proteomics.

[54]  Shizuo Akira,et al.  The RNA helicase RIG-I has an essential function in double-stranded RNA-induced innate antiviral responses , 2004, Nature Immunology.

[55]  B. Li,et al.  Expression profiling reveals off-target gene regulation by RNAi , 2003, Nature Biotechnology.

[56]  R. Juliano,et al.  Evaluating the Specificity of Antisense Oligonucleotide Conjugates , 2002, The Journal of Biological Chemistry.

[57]  T. Tuschl,et al.  Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells , 2001, Nature.

[58]  A. Levin,et al.  Synthetic Oligonucleotides: The Development of Antisense Therapeutics , 1999, Toxicologic pathology.

[59]  R. Carthew,et al.  Use of dsRNA-Mediated Genetic Interference to Demonstrate that frizzled and frizzled 2 Act in the Wingless Pathway , 1998, Cell.

[60]  A. Fire,et al.  Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans , 1998, Nature.

[61]  I. Kerr,et al.  Molecular cloning and characterization of the human double-stranded RNA-activated protein kinase induced by interferon , 1990, Cell.