Targeted Delivery of siRNA

Therapeutic application of siRNA requires delivery to the correct intracellular location, to interact with the RNAi machinery within the target cell, within the target tissue responsible for the pathology. Each of these levels of targeting poses a significant barrier. To overcome these barriers several strategies have been developed, such as chemical modifications of siRNA, viral nucleic acid delivery systems, and nonviral nucleic acid delivery systems. Here, we discuss progress that has been made to improve targeted delivery of siRNA in vivo for each of these strategies.

[1]  A. Lee,et al.  Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

[2]  B. Bosch,et al.  Soluble Receptor-Mediated Targeting of Mouse Hepatitis Coronavirus to the Human Epidermal Growth Factor Receptor , 2005, Journal of Virology.

[3]  Qi Zhou,et al.  Tumor-targeting nanoimmunoliposome complex for short interfering RNA delivery. , 2005, Human gene therapy.

[4]  Satoshi Gojo,et al.  Sonoporation using microbubble BR14 promotes pDNA/siRNA transduction to murine heart. , 2005, Biochemical and biophysical research communications.

[5]  Hongtao Liao,et al.  Biomembrane-permeable and Ribonuclease-resistant siRNA with enhanced activity. , 2005, Oligonucleotides.

[6]  Y. Omidi,et al.  Toxicogenomics of cationic lipid-based vectors for gene therapy: impact of microarray technology. , 2005, Current drug delivery.

[7]  M. Gottesman,et al.  Efficient delivery of RNA interference effectors via in vitro-packaged SV40 pseudovirions. , 2005, Human Gene Therapy.

[8]  Peixuan Guo,et al.  Specific delivery of therapeutic RNAs to cancer cells via the dimerization mechanism of phi29 motor pRNA. , 2005, Human gene therapy.

[9]  Phillip D. Zamore,et al.  Ribo-gnome: The Big World of Small RNAs , 2005, Science.

[10]  T. Ochiya,et al.  Efficient delivery of small interfering RNA to bone-metastatic tumors by using atelocollagen in vivo. , 2005, Proceedings of the National Academy of Sciences of the United States of America.

[11]  J. Seppen,et al.  persistent knockdown of CCR2 function in vivo Lentiviral shRNA silencing of murine bone marrow cell CCR2 leads to , 2013 .

[12]  M. Woodle,et al.  In Vivo Application of RNA Interference: From Functional Genomics to Therapeutics , 2005, Advances in Genetics.

[13]  A. J. Mixson,et al.  Small interfering RNA targeting Raf-1 inhibits tumor growth in vitro and in vivo , 2005, Cancer Gene Therapy.

[14]  P. J. Welch,et al.  State-of-the-art modified RNAi compounds for therapeutics. , 2005, IDrugs : the investigational drugs journal.

[15]  Richard W. Carthew,et al.  Silence from within: Endogenous siRNAs and miRNAs , 2005, Cell.

[16]  M. Sioud.  On the delivery of small interfering RNAs into mammalian cells , 2005, Expert opinion on drug delivery.

[17]  Hideyoshi Harashima,et al.  RNA interference induced by siRNAs modified with 4′‐thioribonucleosides in cultured mammalian cells , 2005, FEBS letters.

[18]  Gregory J. Hannon,et al.  MicroRNA-dependent localization of targeted mRNAs to mammalian P-bodies , 2005, Nature Cell Biology.

[19]  S. Ashley,et al.  RNA interference: a mammalian SID-1 homologue enhances siRNA uptake and gene silencing efficacy in human cells. , 2005, Biochemical and biophysical research communications.

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

[21]  B. Polisky,et al.  Activity of stabilized short interfering RNA in a mouse model of hepatitis B virus replication , 2005, Hepatology.

[22]  R. Griffey,et al.  Positional effect of chemical modifications on short interference RNA activity in mammalian cells. , 2005, Journal of Medicinal Chemistry.

[23]  H. Blau,et al.  Argonaute 2/RISC resides in sites of mammalian mRNA decay known as cytoplasmic bodies , 2005, Nature Cell Biology.

[24]  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.

[25]  F. He,et al.  Cationic lipids enhance siRNA-mediated interferon response in mice. , 2005, Biochemical and biophysical research communications.

[26]  L. Greensmith,et al.  Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model , 2005, Nature Medicine.

[27]  I. Ahmad,et al.  Systemic delivery of RafsiRNA using cationic cardiolipin liposomes silences Raf-1 expression and inhibits tumor growth in xenograft model of human prostate cancer. , 2005, International journal of oncology.

[28]  R. Schiffelers,et al.  Effects of treatment with small interfering RNA on joint inflammation in mice with collagen-induced arthritis. , 2005, Arthritis and rheumatism.

[29]  C. Boshoff,et al.  Inhibiting primary effusion lymphoma by lentiviral vectors encoding short hairpin RNA. , 2005, Blood.

[30]  C. Henderson,et al.  Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS , 2005, Nature Medicine.

[31]  A. Aigner,et al.  RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo , 2005, Gene Therapy.

[32]  G. Nolan,et al.  Gene therapy progress and prospects: Novel gene therapy approaches for AIDS , 2005, Gene Therapy.

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

[34]  D. Palmer,et al.  Helper-dependent adenoviral vectors for gene therapy. , 2005, Human gene therapy.

[35]  Qun Zhou,et al.  Viral vectors for cancer gene therapy: viral dissemination and tumor targeting. , 2005, Current gene therapy.

[36]  Sung Ho Hahm,et al.  Retroviral gene therapy: safety issues and possible solutions. , 2005, Current gene therapy.

[37]  F. Chisari,et al.  Clearance of hepatitis B virus from the liver of transgenic mice by short hairpin RNAs. , 2005, Proceedings of the National Academy of Sciences of the United States of America.

[38]  T. Rana,et al.  Specific and potent RNAi in the nucleus of human cells , 2005, Nature Structural &Molecular Biology.

[39]  F. Porreca,et al.  An efficient intrathecal delivery of small interfering RNA to the spinal cord and peripheral neurons , 2005, Molecular pain.

[40]  W. Denk,et al.  Lentivirus-based genetic manipulations of cortical neurons and their optical and electrophysiological monitoring in vivo , 2004, Proceedings of the National Academy of Sciences of the United States of America.

[41]  M. Manoharan.  RNA interference and chemically modified small interfering RNAs. , 2004, Current opinion in chemical biology.

[42]  Raymond M Schiffelers,et al.  Inhibition of ocular angiogenesis by siRNA targeting vascular endothelial growth factor pathway genes: therapeutic strategy for herpetic stromal keratitis. , 2004, The American journal of pathology.

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

[44]  S. Chae,et al.  Requirement for sphingosine 1-phosphate receptor-1 in tumor angiogenesis demonstrated by in vivo RNA interference. , 2004, The Journal of clinical investigation.

[45]  J. Lieberman,et al.  Small interfering RNA targeting Fas protects mice against renal ischemia-reperfusion injury. , 2004, Proceedings of the National Academy of Sciences of the United States of America.

[46]  S. Ashley,et al.  Systemic siRNA-Mediated Gene Silencing: A New Approach to Targeted Therapy of Cancer , 2004, Annals of surgery.

[47]  T. Rana,et al.  Visualizing a correlation between siRNA localization, cellular uptake, and RNAi in living cells. , 2004, Chemistry & biology.

[48]  K. Taira,et al.  Adenovirus-mediated transfer of siRNA against survivin induced apoptosis and attenuated tumor cell growth in vitro and in vivo. , 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

[49]  Y. Yuzawa,et al.  A Small Interfering RNA Targeting Vascular Endothelial Growth Factor as Cancer Therapeutics , 2004, Cancer Research.

[50]  Anton P. McCaffrey,et al.  In vivo activity of nuclease-resistant siRNAs. , 2004, RNA.

[51]  M. Eccles,et al.  Conjugate for efficient delivery of short interfering RNA (siRNA) into mammalian cells , 2004, FEBS letters.

[52]  E. H. Feinberg,et al.  Transport of dsRNA into Cells by the Transmembrane Protein SID-1 , 2003, Science.

[53]  T. Rana,et al.  siRNA function in RNAi: a chemical modification analysis. , 2003, RNA.

[54]  Robert H. Silverman,et al.  Activation of the interferon system by short-interfering RNAs , 2003, Nature Cell Biology.

[55]  David R Corey,et al.  RNA interference in mammalian cells by chemically-modified RNA. , 2003, Biochemistry.

[56]  A. Klippel,et al.  Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells. , 2003, Nucleic acids research.

[57]  Mouldy Sioud,et al.  Gene silencing by systemic delivery of synthetic siRNAs in adult mice. , 2003, Journal of molecular biology.

[58]  Thomas Tuschl,et al.  Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing. , 2003, Antisense & nucleic acid drug development.

[59]  Jens Kurreck,et al.  Antisense technologies. Improvement through novel chemical modifications. , 2003, European journal of biochemistry.

[60]  P. Monahan,et al.  Safety of adeno-associated virus gene therapy vectors: a current evaluation , 2002, Expert opinion on drug safety.

[61]  J. Kappes,et al.  Safety Considerations in Vector Development , 2001, Somatic cell and molecular genetics.

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

[63]  S. Gambhir,et al.  Imaging chemically modified adenovirus for targeting tumors expressing integrin alphavbeta3 in living mice with mutant herpes simplex virus type 1 thymidine kinase PET reporter gene. , 2006, Journal of nuclear medicine : official publication, Society of Nuclear Medicine.

[64]  J. Wilschut,et al.  Reconstituted influenza virus envelopes as an efficient carrier system for cellular delivery of small-interfering RNAs , 2006, Gene Therapy.

[65]  Tadaharu Tsumoto,et al.  RNAi-induced gene silencing by local electroporation in targeting brain region. , 2005, Journal of neurophysiology.

[66]  H. Mizuguchi,et al.  Gene therapy for human small-cell lung carcinoma by inactivation of Skp-2 with virally mediated RNA interference , 2005, Gene Therapy.

[67]  Raymond M. Schiffelers,et al.  Pharmaceutical Prospects for RNA Interference , 2004, Pharmaceutical Research.

[68]  R. Schiffelers,et al.  Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle. , 2004, Nucleic acids research.

[69]  K. Alexander,et al.  RNA interference using boranophosphate siRNAs: structure-activity relationships. , 2004, Nucleic acids research.

[70]  N. Kosaka,et al.  Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo. , 2004, Nucleic acids research.

[71]  M. Famulok,et al.  Functional tuning of nucleic acids by chemical modifications: tailored oligonucleotides as drugs, devices, and diagnostics. , 2003, Chemical record.

[72]  J. Dausset,et al.  Journal of Biomedicine and Biotechnology , 2001, Journal of biomedicine & biotechnology.