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Review

Cellular uptake and intracellular release are major obstacles to the therapeutic application of siRNA: novel options by phosphorothioate-stimulated delivery

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Pages 1531-1538 | Published online: 05 Oct 2007

Bibliography

  • RANA TM: Illuminating the silence: understanding the structure and function of small RNAs. Nat. Rev. Mol. Cell. Biol. (2007) 8(1):23-36.
  • HANNON GJ: RNA interference. Nature (2002) 418(6894):244-251.
  • ZAMORE PD, HALEY B: Ribo-gnome: the big world of small RNAs. Science (2005) 309(5740):1519-1524.
  • FILIPOWICZ W: RNAi: the nuts and bolts of the RISC machine. Cell (2005) 122(1):17-20.
  • PELLINO JL, JASKIEWICZ L, FILIPOWICZ W et al.: ATP modulates siRNA interactions with an endogenous human Dicer complex. RNA (2005) 11(11):1719-1724.
  • NYKÄNEN A, HALEY B, ZAMORE PD: ATP requirements and small interfering RNA structure in the RNA interference pathway. Cell (2001) 107(3):309-321.
  • MORRIS KV, ROSSI JJ: Lentiviral-mediated delivery of siRNAs for antiviral therapy. Gene Ther. (2006) 13(6):553-558.
  • AMARZGUIOUI M, ROSSI JJ, KIM D: Approaches for chemically synthesized siRNA and vector-mediated RNAi. FEBS Lett. (2005) 579(26):5974-5981.
  • YU JY, DERUITER SL, TURNER DL: RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cells. Proc. Natl. Acad. Sci. USA (2002) 99(9):6047-6052.
  • WIZNEROWICZ M, SZULC J, TRONO D: Tuning silence: conditional systems for RNA interference. Nat. Methods (2006) 3(9):682-688.
  • SNØVE O Jr, ROSSI JJ: Expressing short hairpin RNAs in vivo. Nat. Methods (2006) 3(9):689-695.
  • BERNARDS R, BRUMMELKAMP TR, BEIJERSBERGEN RL: shRNA libraries and their use in cancer genetics. Nat. Methods (2006) 3(9):701-706.
  • GRIMM D, KAY MA: Therapeutic short hairpin RNA expression in the liver: viral targets and vectors. Gene Ther. (2006) 13(6):563-575.
  • HANNON GJ, ROSSI JJ: Unlocking the potential of the human genome with RNA interference. Nature (2004) 431(7006):371-378.
  • DE PAULA D, BENTLEY MV, MAHATO RI: Hydrophobization and bioconjugation for enhanced siRNA delivery and targeting. RNA (2007) 13(4):431-456.
  • DYKXHOORN DM, PALLISER D, LIEBERMAN J: The silent treatment: siRNAs as small molecule drugs. Gene Ther. (2006) 13(6):541-552.
  • BUMCROT D, MANOHARAN M, KOTELIANSKY V et al.: RNAi therapeutics: a potential new class of pharmaceutical drugs. Nat. Chem. Biol. (2006) 2(12):711-719.
  • SANTEL A, ALEKU M, KEIL O et al.: A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium. Gene Ther. (2006) 13(16):1222-1234.
  • LI CX, PARKER A, MENOCAL E et al.: Delivery of RNA interference. Cell Cycle (2006) 5(18):2103-2109.
  • DORSETT Y, TUSCHL T: siRNAs: applications in functional genomics and potential as therapeutics. Nat. Rev. Drug Discov. (2004) 3(4):318-329.
  • KIM DH, ROSSI JJ: Strategies for silencing human disease using RNA interference. Nat. Rev. Genet. (2007) 8(3):173-184.
  • IKEDA Y, TAIRA K: Ligand-targeted delivery of therapeutic siRNA. Pharm. Res. (2006) 23(8):1631-1640.
  • VORNLOCHER HP: Antibody-directed cell-type-specific delivery of siRNA. Trends Mol. Med. (2006) 12(1):1-3.
  • SONG E, ZHU P, LEE SK et al.: Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat. Biotechnol. (2005) 23(6):709-717.
  • URBAN-KLEIN B, WERTH S, ABUHARBEID et al.: RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo. Gene Ther. (2005) 12(5):461-466.
  • SOUTSCHEK J, AKINC A, BRAMLAGE B et al.: Therapeutic silencing of an endogeneous gene by systemic administration of modified siRNAs. Nature (2004) 432(7014):173-178.
  • MINAKUCHI Y, TAKESHITA F, KOSAKA N et al.: Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo. Nucleic Acids Res. (2004) 32(13):E109.
  • MCNAMARA JO II, ANDRECHEK ER, WANG Y et al.: Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras. Nat. Biotechnol. (2006) 24(8):1005-1015.
  • UPRICHARD SL: The therapeutic potential of RNA interference. FEBS Lett. (2005) 579(26):5996-6007.
  • OPALINSKA JB, GEWIRTZ AM: Nucleic-acid therapeutics: basic principles and recent applications. Nat. Rev. Drug Discov. (2002) 1(7):503-514.
  • RETTIG GR, RICE KG: Non-viral gene delivery: from the needle to the nucleus. Expert Opin. Biol. Ther. (2007) 7(6):799-808.
  • WAGNER E: Programmed drug delivery: nanosystems for tumor targeting. Expert Opin. Biol. Ther. (2007) 7(5):587-593.
  • VELDHOEN S, LAUFER SD, TRAMPE A et al.: Cellular delivery of small interfering RNA by a non-covalently attached cell-penetrating peptide: quantitative analysis of uptake and biological effect. Nucleic Acids Res. (2006) 34(22):6561-6573.
  • OVERHOFF M, SCZAKIEL G: Phosphorothioate-stimulated uptake of short interfering RNA by human cells. EMBO Rep. (2005) 6:1176-1181.
  • CONNER SD, SCHMID SL: Regulated portals of entry into the cell. Nature (2003) 422(6927):37-44.
  • BEREZHNA SY, SUPEKOVA L, SUPEK F et al.: siRNA in human cells selectively localizes to target RNA sites. Proc. Natl. Acad. Sci. USA (2006) 103(20):7682-7687.
  • CHIU YL, ALI A, CHU CY et al.: Visualizing a correlation between siRNA localization, cellular uptake, and RNAi in living cells. Chem. Biol. (2004) 11(8):1165-1175.
  • TAGAWA A, MEZZACASA A, HAYER A et al.: Assembly and trafficking of caveolar domains in the cell: caveolae as stable, cargo-triggered, vesicular transporters. J. Cell Biol. (2005) 170(5):769-779.
  • PELKMANS L, BURLI T, ZERIAL M et al.: Caveolin-stabilized membrane domains as multifunctional transport and sorting devices in endocytic membrane traffic. Cell (2004) 118(6):767-780.
  • PARTON RG, RICHARDS AA: Lipid rafts and caveolae as portals for endocytosis: new insight and common mechanisms. Traffic (2003) 4(11):724-738.
  • HUPPI K, MARTIN SE, CAPLEN NJ: Defining and assaying RNAi in mammalian cells. Mol. Cell. (2005) 17(1):1-10.
  • LINGOR P, MICHEL U, SCHÖLL U et al.: Transfection of “naked” siRNA results in endosomal uptake and metabolic impairment in cultured neurons. Biochem. Biophys. Res. Commun. (2004) 315(4):1126-1133.
  • MURATOVSKA A, ECCLES MR: Conjugate for efficient delivery of short interfering RNA (siRNA) into mammalian cells. FEBS Lett. (2004) 558(1-3):63-68.
  • KIMCHI-SARFATY C, BRITTAIN S, GARFIELD S et al.: Efficient delivery of RNA interference effectors via in vitro-packaged SV40 pseudovirions. Hum. Gene Ther. (2005) 16(9):1110-1115.
  • JIANG M, ARZUMANOV AA, GAIT MJ et al.: A bi-functional siRNA construct induces RNA interference and also primes PCR amplification for its own quantification. Nucleic Acids Res. (2005) 33(18):E151.
  • OVERHOFF M, WÜNSCHE W, SCZAKIEL G: Quantitative detection of siRNA and single-stranded oligonucleotides: relationship between uptake and biological activity of siRNA. Nucleic Acids Res. (2004) 32(21):E170.
  • ELBASHIR SM, HARBORTH J, WEBER K et al.: Analysis of gene function in somatic mammalian cells using small interfering RNAs. Methods (2002) 26(2):199-213.
  • ELBASHIR SM, HARBORTH J, LENDECKEL W et al.: Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature (2001) 411(6836):494-498.
  • OVERHOFF M: Untersuchungen zur Struktur-Funktions-Beziehung von small interfering RNA (siRNA) und ein neuartiges Konzept zu ihrer Einschleusung in Säugerzellen. PhD Thesis. University of Luebeck, Germany (2006).
  • LE PU, NABI IR: Distinct caveolae-mediated endocytic pathways target the golgi apparatus and the endoplasmic reticulum. J. Cell Sci. (2003) 116(6):1059-1071.
  • NORKIN LC, ANDERSON HA, WOLFROM SA et al.: Caveolar endocytosis of simian virus 40 is followed by brefeldin A-sensitive transport to the endoplasmic reticulum, where the virus disassembles. J. Virol. (2002) 76(10):5156-5166.
  • MASCHKOWITZ G: Fluoreszenzmikroskopische Untersuchung der Phosphorothioat-stimulierten Aufnahme von small interfering RNA in eukaryotische Zellen. Master Thesis, University of Luebeck (2006).
  • BENNETT FC: General pharmacology of phosphorothioate oligodeoxynucleotides. In: Antisense Drug Technology Principles, Strategies and Applications. Crooke ST (Ed.), Marcel Dekker, Inc., New York, USA (2001):291-318.
  • ROBB GB, BROWN KM, KHURANA J et al.: Specific and potent RNAi in the nucleus of human cells. Nat. Struct. Mol. Biol. (2005) 12(2):133-137.
  • WU H, MACLEOD AR, LIMA WF et al.: Identification and partial purification of human double strand RNase activity. A novel terminating mechanism for oligoribonucleotide antisense drugs. J. Biol. Chem. (1998) 273(5):2532-2542.
  • HORMES R, HOMANN M, OELZE I et al.: The subcellular localization and length of hammerhead ribozymes determine efficacy in human cells. Nucleic Acids Res. (1997) 25(4):769-775.
  • ISOMURA I, MORITA A: Regulation of NF-κB signaling by decoy oligodeoxynucleotides. Microbiol. Immunol. (2006) 50(8):559-563.

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