Cell-type-specific, Aptamer-functionalized Agents for Targeted Disease Therapy

Molecular Therapy - Nucleic Acids - Tập 3 - Trang e169 - 2014
Jiehua Zhou1, John J. Rossi1,2
1Department of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, Duarte, California, USA
2Irell and Manella Graduate School of Biological Sciences, Beckman Research Institute of City of Hope, Duarte, California, USA

Tóm tắt

Từ khóa


Tài liệu tham khảo

Joensuu, 2008, Systemic chemotherapy for cancer: from weapon to treatment, Lancet Oncol, 9, 304, 10.1016/S1470-2045(08)70075-5

Harrison, 2002, Impact of tumor hypoxia and anemia on radiation therapy outcomes, Oncologist, 7, 492, 10.1634/theoncologist.7-6-492

Shapira, 2010, Targeted immunotherapy for colorectal cancer: monoclonal antibodies and immunotoxins, Expert Opin Investig Drugs, 19, S67, 10.1517/13543781003737668

Whitehead, 2009, Knocking down barriers: advances in siRNA delivery, Nat Rev Drug Discov, 8, 129, 10.1038/nrd2742

Langer, 1998, Drug delivery and targeting, Nature, 392, 5

Schrama, 2006, Antibody targeted drugs as cancer therapeutics, Nat Rev Drug Discov, 5, 147, 10.1038/nrd1957

Zhou, 2011, Cell-specific aptamer-mediated targeted drug delivery, Oligonucleotides, 21, 1, 10.1089/oli.2010.0264

Senter, 2001, Selective activation of anticancer prodrugs by monoclonal antibody-enzyme conjugates, Adv Drug Deliv Rev, 53, 247, 10.1016/S0169-409X(01)00206-X

Ercan, 2000, Therapeutic radiopharmaceuticals, Curr Pharm Des, 6, 1085

Allen, 2002, Ligand-targeted therapeutics in anticancer therapy, Nat Rev Cancer, 2, 750, 10.1038/nrc903

Robertson, 1990, Selection in vitro of an RNA enzyme that specifically cleaves single-stranded DNA., Nature, 344, 467, 10.1038/344467a0

Tuerk, 1990, Systematic evolution of ligands by exponential enrichment: RNA ligands to bacteriophage T4 DNA polymerase, Science, 249, 505, 10.1126/science.2200121

Ellington, 1990, In vitro selection of RNA molecules that bind specific ligands, Nature, 346, 818, 10.1038/346818a0

Tan, 2011, Molecular aptamers for drug delivery, Trends Biotechnol, 29, 634, 10.1016/j.tibtech.2011.06.009

Mayer, 2009, The chemical biology of aptamers, Angew Chem Int Ed Engl, 48, 2672, 10.1002/anie.200804643

Blank, 2005, Aptamers as tools for target validation, Curr Opin Chem Biol., 9, 336, 10.1016/j.cbpa.2005.06.011

Sundaram, 2013, Therapeutic RNA aptamers in clinical trials, Eur J Pharm Sci, 48, 259, 10.1016/j.ejps.2012.10.014

Reinemann, 2014, Aptamer-modified nanoparticles and their use in cancer diagnostics and treatment, Swiss Med Wkly, 144, w13908

Bompiani, 2012, Antidote control of aptamer therapeutics: the road to a safer class of drug agents, Curr Pharm Biotechnol, 13, 1924, 10.2174/138920112802273137

Younas, 2010, HMG-CoA reductase inhibitors in kidney transplant recipients receiving tacrolimus: statins not associated with improved patient or graft survival, BMC Nephrol, 11, 5, 10.1186/1471-2369-11-5

Kulbachinskiy, 2007, Methods for selection of aptamers to protein targets, Biochemistry (Mosc), 72, 1505, 10.1134/S000629790713007X

Guo, 2008, CELL-SELEX: Novel perspectives of aptamer-based therapeutics, Int J Mol Sci, 9, 668, 10.3390/ijms9040668

Chen, 2010, Mutations in the Salmonella enterica serovar Choleraesuis cAMP-receptor protein gene lead to functional defects in the SPI-1 Type III secretion system, Vet Res., 41, 5, 10.1051/vetres/2009053

Berezovski, 2005, Nonequilibrium capillary electrophoresis of equilibrium mixtures: a universal tool for development of aptamers, J Am Chem Soc, 127, 3165, 10.1021/ja042394q

Berezovski, 2006, Non-SELEX selection of aptamers, J Am Chem Soc, 128, 1410, 10.1021/ja056943j

Mallikaratchy, 2006, Selection of DNA ligands for protein kinase C-delta, Chem Commun (Camb), 3229, 10.1039/b604778e

Farokhzad, 2005, Microfluidic system for studying the interaction of nanoparticles and microparticles with cells, Anal Chem., 77, 5453, 10.1021/ac050312q

Lupold, 2002, Identification and characterization of nuclease-stabilized RNA molecules that bind human prostate cancer cells via the prostate-specific membrane antigen, Cancer Res., 62, 4029

Kraus, 1998, Cutting edge: novel RNA ligands able to bind CD4 antigen and inhibit CD4+ T lymphocyte function, J Immunol, 160, 5209, 10.4049/jimmunol.160.11.5209

Zhou, 2009, Selection, characterization and application of new RNA HIV gp 120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells, Nucleic Acids Res., 37, 3094, 10.1093/nar/gkp185

Zhou, 2013, Dual functional BAFF receptor aptamers inhibit ligand-induced proliferation and deliver siRNAs to NHL cells, Nucleic Acids Res., 41, 4266, 10.1093/nar/gkt125

Li, 2010, Directed evolution of gold nanoparticle delivery to cells, Chem Commun (Camb), 46, 392, 10.1039/B920865H

Cerchia, 2010, Targeting cancer cells with nucleic acid aptamers, Trends Biotechnol, 28, 517, 10.1016/j.tibtech.2010.07.005

Cerchia, 2005, Neutralizing aptamers from whole-cell SELEX inhibit the RET receptor tyrosine kinase, PLoS Biol., 3, e123, 10.1371/journal.pbio.0030123

Pestourie, 2006, Comparison of different strategies to select aptamers against a transmembrane protein target, Oligonucleotides, 16, 323, 10.1089/oli.2006.16.323

Fang, 2010, Aptamers generated from cell-SELEX for molecular medicine: a chemical biology approach, Acc Chem Res., 43, 48, 10.1021/ar900101s

Shangguan, 2006, Aptamers evolved from live cells as effective molecular probes for cancer study, Proc Natl Acad Sci USA., 103, 11838, 10.1073/pnas.0602615103

Shangguan, 2007, Aptamers evolved from cultured cancer cells reveal molecular differences of cancer cells in patient samples, Clin Chem., 53, 1153, 10.1373/clinchem.2006.083246

Thiel, 2012, Delivery of chemo-sensitizing siRNAs to HER2+-breast cancer cells using RNA aptamers, Nucleic Acids Res., 40, 6319, 10.1093/nar/gks294

Ohuchi, 2012, Cell-SELEX Technology, Biores Open Access, 1, 265, 10.1089/biores.2012.0253

Mi, 2010, In vivo selection of tumor-targeting RNA motifs, Nat Chem Biol., 6, 22, 10.1038/nchembio.277

Cheng, 2013, In vivo SELEX for identification of brain-penetrating aptamers, Mol Ther Nucleic Acids, 2, e67, 10.1038/mtna.2012.59

Keefe, 2008, SELEX with modified nucleotides, Curr Opin Chem Biol., 12, 448, 10.1016/j.cbpa.2008.06.028

Ray, 2013, Application of aptamers for targeted therapeutics, Arch Immunol Ther Exp, 61, 255, 10.1007/s00005-013-0227-0

Xing, 2012, DNA aptamer functionalized nanomaterials for intracellular analysis, cancer cell imaging and drug delivery, Curr Opin Chem Biol., 16, 429, 10.1016/j.cbpa.2012.03.016

Keefe, 2010, Aptamers as therapeutics, Nat Rev Drug Discov, 9, 537, 10.1038/nrd3141

Shu, 2014, Stable RNA nanoparticles as potential new generation drugs for cancer therapy, Adv Drug Deliv Rev, 66C, 74, 10.1016/j.addr.2013.11.006

Zhou, 2009, The therapeutic potential of cell-internalizing aptamers, Curr Top Med Chem., 9, 1144, 10.2174/156802609789630893

Bourrinet, 2006, Preclinical safety and pharmacokinetic profile of ferumoxtran-10, an ultrasmall superparamagnetic iron oxide magnetic resonance contrast agent, Invest Radiol, 41, 313, 10.1097/01.rli.0000197669.80475.dd

Kim, 2012, T Cell immunoglobulin mucin domain (TIM)-3 promoter activity in a human mast cell line, Immune Netw, 12, 207, 10.4110/in.2012.12.5.207

Piao, 2013, Density dependence across multiple life stages in a temperate old-growth forest of northeast China, Oecologia, 172, 207, 10.1007/s00442-012-2481-y

Fire, 1998, Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans, Nature, 391, 806, 10.1038/35888

Davidson, 2011, Current prospects for RNA interference-based therapies, Nat Rev Genet, 12, 329, 10.1038/nrg2968

Lares, 2010, RNAi and small interfering RNAs in human disease therapeutic applications, Trends Biotechnol, 28, 570, 10.1016/j.tibtech.2010.07.009

de Fougerolles, 2007, Interfering with disease: a progress report on siRNA-based therapeutics, Nat Rev Drug Discov, 6, 443, 10.1038/nrd2310

Castanotto, 2009, The promises and pitfalls of RNA-interference-based therapeutics, Nature, 457, 426, 10.1038/nature07758

McNamara 2nd, 2006, Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras, Nat Biotechnol, 24, 1005, 10.1038/nbt1223

Dassie, 2009, Systemic administration of optimized aptamer-siRNA chimeras promotes regression of PSMA-expressing tumors, Nat Biotechnol, 27, 839, 10.1038/nbt.1560

Pastor, 2010, Induction of tumour immunity by targeted inhibition of nonsense-mediated mRNA decay, Nature, 465, 227, 10.1038/nature08999

Zhou, 2008, Novel dual inhibitory function aptamer-siRNA delivery system for HIV-1 therapy, Mol Ther, 16, 1481, 10.1038/mt.2008.92

Neff, 2011, An aptamer-siRNA chimera suppresses HIV-1 viral loads and protects from helper CD4(+) T cell decline in humanized mice, Sci Transl Med, 3, 66ra66, 10.1126/scitranslmed.3001581

Wheeler, 2011, Inhibition of HIV transmission in human cervicovaginal explants and humanized mice using CD4 aptamer-siRNA chimeras, J Clin Invest, 121, 2401, 10.1172/JCI45876

Ni, 2011, Prostate-targeted radiosensitization via aptamer-shRNA chimeras in human tumor xenografts, J Clin Invest, 121, 2383, 10.1172/JCI45109

Wullner, 2008, Cell-specific induction of apoptosis by rationally designed bivalent aptamer-siRNA transcripts silencing eukaryotic elongation factor 2, Curr Cancer Drug Targets, 8, 554, 10.2174/156800908786241078

Zhu, 2012, Inhibition of HIV-1 protease expression in T cells owing to DNA aptamer-mediated specific delivery of siRNA, Eur J Med Chem., 56, 396, 10.1016/j.ejmech.2012.07.045

Mu, 2013, Solubilization of flurbiprofen into aptamer-modified PEG-PLA micelles for targeted delivery to brain-derived endothelial cells in vitro, J Microencapsul, 30, 701, 10.3109/02652048.2013.778907

Sun, 2010, Insulin like growth factor-1 prevents 1-mentyl-4-phenylphyridinium-induced apoptosis in PC12 cells through activation of glycogen synthase kinase-3beta, Toxicology, 271, 5, 10.1016/j.tox.2010.01.001

Altschuler, 2000, Clathrin-mediated endocytosis of MUC1 is modulated by its glycosylation state, Mol Biol Cell, 11, 819, 10.1091/mbc.11.3.819

Gendler, 2001, MUC1, the renaissance molecule, J Mammary Gland Biol Neoplasia, 6, 339, 10.1023/A:1011379725811

Xu, 2013, Aptamer-conjugated and doxorubicin-loaded unimolecular micelles for targeted therapy of prostate cancer, Biomaterials, 34, 5244, 10.1016/j.biomaterials.2013.03.006

Kotula, 2012, Aptamer-mediated delivery of splice-switching oligonucleotides to the nuclei of cancer cells, Nucleic Acid Ther, 22, 187, 10.1089/nat.2012.0347

Bates, 2009, Discovery and development of the G-rich oligonucleotide AS1411 as a novel treatment for cancer, Exp Mol Pathol, 86, 151, 10.1016/j.yexmp.2009.01.004

Bates, 1999, Antiproliferative activity of G-rich oligonucleotides correlates with protein binding, J Biol Chem., 274, 26369, 10.1074/jbc.274.37.26369

Chu, 2006, Aptamer mediated siRNA delivery, Nucleic Acids Res., 34, e73, 10.1093/nar/gkl388

Zhou, 2013, Functional in vivo delivery of multiplexed anti-HIV-1 siRNAs via a chemically synthesized aptamer with a sticky bridge, Mol Ther, 21, 192, 10.1038/mt.2012.226

Wu, 2013, Nucleolin targeting AS1411 modified protein nanoparticle for antitumor drugs delivery, Mol Pharm, 10, 3555, 10.1021/mp300686g

Gilboa, 2013, Use of oligonucleotide aptamer ligands to modulate the function of immune receptors, Clin Cancer Res., 19, 1054, 10.1158/1078-0432.CCR-12-2067

Pastor, 2011, Targeting 4-1BB costimulation to disseminated tumor lesions with bi-specific oligonucleotide aptamers, Mol Ther, 19, 1878, 10.1038/mt.2011.145

McNamara, 2008, Multivalent 4-1BB binding aptamers costimulate CD8+ T cells and inhibit tumor growth in mice, J Clin Invest, 118, 376, 10.1172/JCI33365

Huang, 2009, Molecular assembly of an aptamer-drug conjugate for targeted drug delivery to tumor cells, ChembioChem., 10, 862, 10.1002/cbic.200800805

Xiao, 2008, Cell-specific internalization study of an aptamer from whole cell selection, Chemistry, 14, 1769, 10.1002/chem.200701330

Gao, 2012, Whole-cell SELEX aptamer-functionalised poly(ethyleneglycol)-poly(epsilon-caprolactone) nanoparticles for enhanced targeted glioblastoma therapy, Biomaterials, 33, 6264, 10.1016/j.biomaterials.2012.05.020

Chu, 2006, Aptamer:toxin conjugates that specifically target prostate tumor cells, Cancer Res., 66, 5989, 10.1158/0008-5472.CAN-05-4583

Chen, 2008, Aptamer-based endocytosis of a lysosomal enzyme, Proc Natl Acad Sci USA., 105, 15908, 10.1073/pnas.0808360105

Bagalkot, 2006, An aptamer-doxorubicin physical conjugate as a novel targeted drug-delivery platform, Angew Chem Int Ed Engl, 45, 8149, 10.1002/anie.200602251

Hu, 2012, Novel MUC1 aptamer selectively delivers cytotoxic agent to cancer cells in vitro, PLoS One, 7, e31970, 10.1371/journal.pone.0031970

Liu, 2012, Novel HER2 aptamer selectively delivers cytotoxic drug to HER2-positive breast cancer cells in vitro, J Transl Med, 10, 148, 10.1186/1479-5876-10-148

Tan, 2012, Designer tridentate mucin 1 aptamer for targeted drug delivery, J Pharm Sci, 101, 1672, 10.1002/jps.23101

Shieh, 2010, Aptamer-based tumor-targeted drug delivery for photodynamic therapy, ACS Nano, 4, 1433, 10.1021/nn901374b

Rana, 2012, Monolayer coated gold nanoparticles for delivery applications, Adv Drug Deliv Rev, 64, 200, 10.1016/j.addr.2011.08.006

Luo, 2011, Release of photoactivatable drugs from plasmonic nanoparticles for targeted cancer therapy, ACS Nano, 5, 7796, 10.1021/nn201592s

Wang, 2012, Assembly of aptamer switch probes and photosensitizer on gold nanorods for targeted photothermal and photodynamic cancer therapy, ACS Nano, 6, 5070, 10.1021/nn300694v

Zhao, 2013, An ultra pH-sensitive and aptamer-equipped nanoscale drug-delivery system for selective killing of tumor cells, Small, 9, 3477, 10.1002/smll.201202694

Wang, 2008, Superparamagnetic iron oxide nanoparticle-aptamer bioconjugates for combined prostate cancer imaging and therapy, ChemMedChem., 3, 1311, 10.1002/cmdc.200800091

Meng, 2012, Single walled carbon nanotubes as drug delivery vehicles: targeting doxorubicin to tumors, Biomaterials, 33, 1689, 10.1016/j.biomaterials.2011.11.004

Taghdisi, 2011, Reversible targeting and controlled release delivery of daunorubicin to cancer cells by aptamer-wrapped carbon nanotubes, Eur J Pharm Biopharm, 77, 200, 10.1016/j.ejpb.2010.12.005

Zhu, 2008, Regulation of singlet oxygen generation using single-walled carbon nanotubes, J Am Chem Soc, 130, 10856, 10.1021/ja802913f

Yang, 2012, Near-infrared light-triggered, targeted drug delivery to cancer cells by aptamer gated nanovehicles, Adv Mater, 24, 2890, 10.1002/adma.201104797

Kim, 2012, Molecular imaging of a cancer-targeting theragnostics probe using a nucleolin aptamer- and microRNA-221 molecular beacon-conjugated nanoparticle, Biomaterials, 33, 207, 10.1016/j.biomaterials.2011.09.023

Savla, 2011, Tumor targeted quantum dot-mucin 1 aptamer-doxorubicin conjugate for imaging and treatment of cancer, J Control Release, 153, 16, 10.1016/j.jconrel.2011.02.015

Shen, 2012, Nanovector delivery of siRNA for cancer therapy, Cancer Gene Ther, 19, 367, 10.1038/cgt.2012.22

Kang, 2010, A liposome-based nanostructure for aptamer directed delivery, Chem Commun (Camb), 46, 249, 10.1039/B916911C

Cao, 2009, Reversible cell-specific drug delivery with aptamer-functionalized liposomes, Angew Chem Int Ed Engl, 48, 6494, 10.1002/anie.200901452

Wilner, 2012, An RNA alternative to human transferrin: a new tool for targeting human cells, Mol Ther Nucleic Acids, 1, e21, 10.1038/mtna.2012.14

Wu, 2013, Engineering of switchable aptamer micelle flares for molecular imaging in living cells, ACS Nano, 7, 5724, 10.1021/nn402517v

Wu, 2010, DNA aptamer-micelle as an efficient detection/delivery vehicle toward cancer cells, Proc Natl Acad Sci USA., 107, 5, 10.1073/pnas.0909611107

Zhao, 2012, Prodrug strategy for PSMA-targeted delivery of TGX-221 to prostate cancer cells, Mol Pharm, 9, 1705, 10.1021/mp3000309

Cohen, 2013, Targeted in vitro photodynamic therapy via aptamer-labeled, porphyrin-loaded virus capsids, J Photochem Photobiol B, 121, 67, 10.1016/j.jphotobiol.2013.02.013

Liu, 2013, A universal protein tag for delivery of SiRNA-aptamer chimeras, Sci Rep, 3, 3129, 10.1038/srep03129

Li, 2013, Smart ligand: aptamer-mediated targeted delivery of chemotherapeutic drugs and siRNA for cancer therapy, J Control Release, 171, 152, 10.1016/j.jconrel.2013.06.006

Farokhzad, 2004, Nanoparticle-aptamer bioconjugates: a new approach for targeting prostate cancer cells, Cancer Res., 64, 7668, 10.1158/0008-5472.CAN-04-2550

Farokhzad, 2006, Targeted nanoparticle-aptamer bioconjugates for cancer chemotherapy in vivo, Proc Natl Acad Sci USA., 103, 6315, 10.1073/pnas.0601755103

Dhar, 2008, Targeted delivery of cisplatin to prostate cancer cells by aptamer functionalized Pt(IV) prodrug-PLGA-PEG nanoparticles, Proc Natl Acad Sci USA., 105, 17356, 10.1073/pnas.0809154105

Gu, 2008, Precise engineering of targeted nanoparticles by using self-assembled biointegrated block copolymers, Proc Natl Acad Sci USA., 105, 2586, 10.1073/pnas.0711714105

Cheng, 2007, Formulation of functionalized PLGA-PEG nanoparticles for in vivo targeted drug delivery, Biomaterials, 28, 869, 10.1016/j.biomaterials.2006.09.047

Aravind, 2012, AS1411 aptamer tagged PLGA-lecithin-PEG nanoparticles for tumor cell targeting and drug delivery, Biotechnol Bioeng, 109, 2920, 10.1002/bit.24558

Zhao, 2011, A nanocomplex that is both tumor cell-selective and cancer gene-specific for anaplastic large cell lymphoma, J Nanobiotechnology, 9, 2, 10.1186/1477-3155-9-2

Morris, 1994, Fusion of a kinase gene, ALK, to a nucleolar protein gene, NPM, in non-Hodgkin's lymphoma, Science, 263, 1281, 10.1126/science.8122112

Yang, 2012, Prostate-targeted biodegradable nanoparticles loaded with androgen receptor silencing constructs eradicate xenograft tumors in mice, Nanomedicine (Lond), 7, 1297, 10.2217/nnm.12.14

Bagalkot, 2011, siRNA-aptamer chimeras on nanoparticles: preserving targeting functionality for effective gene silencing, ACS Nano, 5, 8131, 10.1021/nn202772p

Boas, 2004, Dendrimers in drug research, Chem Soc Rev, 33, 43, 10.1039/b309043b

Lee, 2011, Targeted chemoimmunotherapy using drug-loaded aptamer-dendrimer bioconjugates, J Control Release, 155, 435, 10.1016/j.jconrel.2011.05.025

Wu, 2011, Second-generation aptamer-conjugated PSMA-targeted delivery system for prostate cancer therapy, Int J Nanomedicine, 6, 1747

Zhang, 2007, Co-delivery of hydrophobic and hydrophilic drugs from nanoparticle-aptamer bioconjugates, Chem Med Chem., 2, 1268, 10.1002/cmdc.200700121

Kim, 2010, Prostate cancer cell death produced by the co-delivery of Bcl-xL shRNA and doxorubicin using an aptamer-conjugated polyplex, Biomaterials, 31, 4592, 10.1016/j.biomaterials.2010.02.030

Ng, 2006, Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease, Nat Rev Drug Discov, 5, 123, 10.1038/nrd1955

Tucker, 1999, Detection and plasma pharmacokinetics of an anti-vascular endothelial growth factor oligonucleotide-aptamer (NX1838) in rhesus monkeys, J Chromatogr B Biomed Sci Appl, 732, 203, 10.1016/S0378-4347(99)00285-6

Drolet, 2000, Pharmacokinetics and safety of an anti-vascular endothelial growth factor aptamer (NX1838) following injection into the vitreous humor of rhesus monkeys, Pharm Res., 17, 1503, 10.1023/A:1007657109012