The DOI matches its Crossref record; 241 of the 243 references it cites were found in a registry or an index.
Progress in Antisense Technology
Abstract
Antisense technology exploits oligonucleotide analogs to bind to target RNAs via Watson-Crick hybridization. Once bound, the antisense agent either disables or induces the degradation of the target RNA. Antisense agents can also alter splicing. During the past decade, much has been learned about the basic mechanisms of antisense, the medicinal chemistry, and the pharmacologic, pharmacokinetic, and toxicologic properties of antisense molecules. Antisense technology has proven valuable in gene functionalization and target validation. With one drug marketed, Vitravenetm, and approximately 20 antisense drugs in clinical development, it appears that antisense drugs may prove important in the treatment of a wide range of diseases.
Overview
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
In the citing paper
“Though fat accumulation, and specifically diacylglycerol accumulation, was equal in all groups, the PKCε antisense oligonucleotide improved hepatic insulin sensitivity and insulin signalling.”
From Lipid-induced insulin resistance: unravelling the mechanism (2010) · cites this paper as [107]
Citations
Cites
Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
Elbashir, S. M., Harborth, J., Lendeckel, W., Yalcin, A., Weber, K., & Tuschl, T. (2001) · Nature · doi:10.1038/35078107 · Cited by 9,456
CpG motifs in bacterial DNA trigger direct B-cell activation
Krieg, A. M., Yi, A.-K., Matson, S., Waldschmidt, T. J., Bishop, G. A., Teasdale, R., Koretzky, G. A., & Klinman, D. M. (1995) · Nature · doi:10.1038/374546a0 · Cited by 3,508
Zamore, P. D., Tuschl, T., Sharp, P. A., & Bartel, D. P. (2000) · Cell · doi:10.1016/s0092-8674(00)80620-0 · Cited by 2,751
Severe Psoriasis – Oral Therapy with a New Retinoid
Fredriksson, T., & Pettersson, U. (2009) · Dermatologica · doi:10.1159/000250839 · Cited by 2,718
Predicting DNA duplex stability from the base sequence
Breslauer, K. J., Frank, R., Blöcker, H., & Marky, L. A. (1986) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.83.11.3746 · Cited by 1,837
Zamecnik, P. C., & Stephenson, M. L. (1978) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.75.1.280 · Cited by 1,792
Functional genomic analysis of C. elegans chromosome I by systematic RNA interference
Fraser, A. G., Kamath, R. S., Zipperlen, P., Martinez-Campos, M., Sohrmann, M., & Ahringer, J. (2000) · Nature · doi:10.1038/35042517 · Cited by 1,761
SISSAOUI, M. (2018) · Archives of Clinical Microbiology · doi:10.4172/1989-8436-c3-011 · Cited by 1,352
Antisense Oligonucleotides as Therapeutic Agents—Is the Bullet Really Magical?
Stein, C., & Cheng, Y. C. (1993) · Science · doi:10.1126/science.8351515 · Cited by 1,300
Morpholino Antisense Oligomers: Design, Preparation, and Properties
Summerton, J. E., & Weller, D. D. (1997) · Antisense and Nucleic Acid Drug Development · doi:10.1089/oli.1.1997.7.187 · Cited by 1,137
Research works printed but not linked: 39
- Crouch RJ, 1985, Nucleases, 211
- Morvan F, 1991, Anticancer Drug Des., 6, 521
- Mirabelli CK, 1991, Anti-Cancer Drug Design, 6, 647
- 42. Cohen JS. 1993. Phosphorothioate oligodeoxynucleotides. See Ref.42a, pp.205–22
- Crooke ST, 1996, J. Pharmacol. Exp. Ther., 277, 923
- Joos RW, 1969, J. Pharmacol. Exp. Ther., 166, 113
- Crooke RM, 1995, J. Pharmacol. Exp. Ther., 275, 462
- Gao W-Y, 1992, Mol. Pharmacol., 41, 223
- Stein CA, 1991, J. Acquired Immune Deficiency Syndromes, 4, 686
- Cossum PA, 1993, J. Pharmacol. Exp. Ther., 267, 1181
- Crooke ST, 1993, Curr. Opin. Invest. Drugs, 2, 1045
- Sands H, 1995, Mol. Pharmacol., 47, 636
- Crooke RM, 1991, Anti-Cancer Drug Design, 6, 609
- 67. Crooke RM. 1993. in vitro and in vivo toxicology of first generation analogs. See Ref.42a, pp.471–92
- Bennett CF, 1992, Mol. Pharmacol., 41, 1023
- Iversen P, 1992, Anticancer Drug Des., 6, 531
- Cossum PA, 1994, J. Pharmacol. Exp. Ther., 269, 89
- 10.1038/clpt.1994.189 · DOI
- Butler M, 1997, Lab. Invest., 77, 379
- Sands H, 1994, Mol. Pharmacol., 45, 932
- Graham MJ, 1998, J. Pharmacol. Exp. Ther., 286, 447
- 95. Mirabelli CK, Crooke ST. 1993. Antisense oligonucleotides in the context of modern molecular drug discovery and development. See Ref.42a, pp.7–35
- 103. Crooke ST, Mirabelli CK. 1993. Antisense oligonucleotides in the context of modern molecular drug discovery and development. See Ref.42a, pp.7–35
- Zhang S-P, 1994, J. Pharmacol. Exp. Ther., 271, 1462
- Dean N, 1996, Cancer Res., 56, 3499
- Maksymowych W, 1999, Arthritis Rheumatol., 42, S170
- O'Dwyer PJ, 1999, Clin. Cancer Res., 5, 3977
- Henry SP, 1997, J. Pharmacol. Exp. Ther., 281, 810
- Cornish KG, 1993, Pharmacol.Comm., 3, 239
- Henry SP, 1997, Anti-Cancer Drug Des., 12, 395
- Wallace TL, 1996, J. Pharmacol. Exp. Ther., 278, 1306
- Henry S, 1994, Pharm. Res., 11, S-353/S1-S490
- Lemonidis-Farrar K, 2000, Toxicologist, 42, 120
- Yuen AR, 1999, Clin. Cancer Res., 5, 3357
- 164. Crooke ST. 2001. Basic principles. See Ref.46a, pp.1–28
- Zellweger T, 2001, J. Pharmacol. Exp. Ther., 298, 1
- Geary RS, 2001, J. Pharmacol. Exp. Ther., 296, 898
- Geary RS, 2001, J. Pharmacol. Exp. Ther., 296, 890
- Henry SP, 2000, J. Pharmacol. Exp. Ther., 292, 468
Cited by
Sorted by citations
Lipid-induced insulin resistance: unravelling the mechanism
Samuel, V. T., Petersen, K. F., & Shulman, G. I. (2010) · The Lancet · doi:10.1016/s0140-6736(10)60408-4 · Cited by 1,189
Chemistry, structure and function of approved oligonucleotide therapeutics
Egli, M., & Manoharan, M. (2023) · Nucleic Acids Research · doi:10.1093/nar/gkad067 · Cited by 909
Progress towards in Vivo Use of siRNAs
Behlke, M. A. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.01.001 · Cited by 677
“Currently, over 20 antisense drugs are in various stages of clinical testing [12].”
BackgroundObesity, adiposity, and dyslipidemia: a consensus statement from the National Lipid Association
Bays, H. E., Toth, P. P., Kris-Etherton, P. M., Abate, N., Aronne, L. J., Brown, W. V., Gonzalez-Campoy, J. M., Jones, S. R., Kumar, R., La Forge, R., et al. (2013) · Journal of Clinical Lipidology · doi:10.1016/j.jacl.2013.04.001 · Cited by 521
“ASOs are synthetic oligonucleotides modified with a 20-O-(2-methoxy) ethyl modification and phosphorothioate bond to enhance potency, stability, and cell permeability.(49) They are taken up preferentially in liver, adipose tissue, and the kidney, although not in other key tissues such as muscle, brain, or pancreatic b…”
BackgroundAlternative splicing in disease and therapy
Garcia-Blanco, M. A., Baraniak, A. P., & Lasda, E. L. (2004) · Nature Biotechnology · doi:10.1038/nbt964 · Cited by 527
“Oligonucleotide-mediated therapies Gene-specific therapy can be mediated by oligonucleotides or oligonucleotide-like compounds, some of which have been shown to have clinical effectiveness in other application”
BackgroundLong non-coding RNAs: From disease code to drug role
Chen, Y., Li, Z., Chen, X., & Zhang, S. (2020) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2020.10.001 · Cited by 515
Antisense oligonucleotide therapy for neurodegenerative disease
Smith, R. A. (2006) · Journal of Clinical Investigation · doi:10.1172/jci25424 · Cited by 506
“Exploiting this and the enhanced tolerability and potency of second-generation oligonucleotides (9) and the fact that drug administration by direct pumping into the CSF is routinely done in humans (11, 12), we describe herein an effective, dosage-regulatable method of gene therapy capable of…”
BackgroundMethodChemical Modification of siRNAs for In Vivo Use
Behlke, M. A. (2008) · Oligonucleotides · doi:10.1089/oli.2008.0164 · Cited by 464
“A variety of chemically modifi ed antisense compounds have been successfully used for in vivo experimental biological studies and over a dozen candidate compounds are in various stages of clinical testing for use as therapeutic agents for a variety of disease indications (Crooke, 2004).”
Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides
Juliano, R. L., Alam, M. R., Dixit, V., & Kang, H. M. (2008) · Nucleic Acids Research · doi:10.1093/nar/gkn342 · Cited by 442
ANTISENSE OLIGONUCLEOTIDES: FROM DESIGN TO THERAPEUTIC APPLICATION
Chan, J. H. P., Lim, S., & Wong, W. S. F. (2006) · Clinical and Experimental Pharmacology and Physiology · doi:10.1111/j.1440-1681.2006.04403.x · Cited by 443
“ASO and location of hybridization, resulting in reduced levels of translation of the target transcript.(1) The ASO is not only a useful tool for studies of loss-of-gene function and target validation, but also highly valuable as a novel therapeutic strategy to treat any disease that is linked to dysregulated gene expr…”
BackgroundInfluentialFung, G., Luo, H., Qiu, Y., Yang, D., & McManus, B. (2016) · Circulation Research · doi:10.1161/circresaha.115.306573 · Cited by 444
Molecular Mechanisms of Antisense Oligonucleotides
Crooke, S. T. (2017) · Nucleic Acid Therapeutics · doi:10.1089/nat.2016.0656 · Cited by 415
RNA interference: From gene silencing to gene-specific therapeutics
Leung, R. K. M., & Whittaker, P. A. (2005) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2005.03.004 · Cited by 391
“For over a decade now, targeting mRNA for both target validation and as a therapeutic strategy has been actively pursued using ribozymes (Breaker, 2004) or antisense (Crooke, 2004) approaches.”
BackgroundInfluentialNatural and engineered nucleic acids as tools to explore biology
Breaker, R. R. (2004) · Nature · doi:10.1038/nature03195 · Cited by 363
“For example, antisense oligonucleotides (whose sequences are complementary to their target genes) have been developed to selectively inhibit a variety of gene”
BackgroundKastelein, J. J. P., Wedel, M. K., Baker, B. F., Su, J. Q., Bradley, J. D., Yu, R. Z., Chuang, E., Graham, M. J., & Crooke, R. M. (2006) · Circulation · doi:10.1161/circulationaha.105.606442 · Cited by 357
“Constipation 1 (14) 0 0 7 (78) 0 7 (24)”
siRNA-based approaches in cancer therapy
Devi, G. R. (2006) · Cancer Gene Therapy · doi:10.1038/sj.cgt.7700931 · Cited by 359
RNA Interference: From Basic Research to Therapeutic Applications
Kurreck, J. (2009) · Angewandte Chemie International Edition · doi:10.1002/anie.200802092 · Cited by 347
Diacylglycerol Activation of Protein Kinase Cε and Hepatic Insulin Resistance
Jornayvaz, F. R., & Shulman, G. I. (2012) · Cell Metabolism · doi:10.1016/j.cmet.2012.03.005 · Cited by 331
Biological Barriers to Therapy with Antisense and siRNA Oligonucleotides
Juliano, R. L., Bauman, J. A., Kang, H. M., & Ming, X. (2009) · Molecular Pharmaceutics · doi:10.1021/mp900093r · Cited by 306
Nano-flares for mRNA Regulation and Detection
Prigodich, A. E., Seferos, D. S., Massich, M. D., Giljohann, D. A., Lane, B. C., & Mirkin, C. A. (2009) · ACS Nano · doi:10.1021/nn9003814 · Cited by 284
“In previous work using similar nanoconjugates, we observed depletion in protein levels,(45) which can be caused by either translation inhibition or mRNA depletion.(37) Here, we find that nanoconjugates deplete mRNA, a phenomenon that has been observed with traditional antisense oligonucleotides.”
BackgroundFructose induced lipogenesis: from sugar to fat to insulin resistance
Samuel, V. T. (2010) · Trends in Endocrinology and Metabolism · doi:10.1016/j.tem.2010.10.003 · Cited by 271
Zhou, W., Ding, J., & Liu, J. (2017) · Theranostics · doi:10.7150/thno.17736 · Cited by 253
Recent developments in oligonucleotide conjugation
Singh, Y., Murat, P., & Defrancq, É. (2010) · Chemical Society Reviews · doi:10.1039/b911431a · Cited by 263
The involvement of microRNAs in neurodegenerative diseases
Maciotta, S., Meregalli, M., & Torrente, Y. (2013) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2013.00265 · Cited by 260
“To avoid the action of nucleases, chemical modification or non-viral carriers can be used (Borchard, 2001; Wang et al., 2002; Crooke, 2004; Juliano, 2005; Juliano et al., 2008).”
MethodTherapeutic potential of nanoparticulate systems for macrophage targeting
Chellat, F., Merhi, Y., Moreau, A., & Yahia, L. (2005) · Biomaterials · doi:10.1016/j.biomaterials.2005.05.044 · Cited by 237
“[144]).”
Advances in treating amyotrophic lateral sclerosis: insights from pathophysiological studies
Vucic, S., Rothstein, J. D., & Kiernan, M. C. (2014) · Trends in Neurosciences · doi:10.1016/j.tins.2014.05.006 · Cited by 233
PEG-Modified Carbon Nanotubes in Biomedicine: Current Status and Challenges Ahead
Bottini, M., Rosato, N., & Bottini, N. (2011) · Biomacromolecules · doi:10.1021/bm201020h · Cited by 220
Design of a small molecule against an oncogenic noncoding RNA
Velagapudi, S. P., Cameron, M. D., Haga, C. L., Rosenberg, L. H., Lafitte, M., Duckett, D. R., Phinney, D. G., & Disney, M. D. (2016) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1523975113 · Cited by 225
Retinaldehyde dehydrogenase 1 regulates a thermogenic program in white adipose tissue
Kiefer, F. W., Vernochet, C., O’Brien, P., Spoerl, S., Brown, J. D., Nallamshetty, S., Zeyda, M., Stulnig, T. M., Cohen, D. E., Kahn, C. R., et al. (2012) · Nature Medicine · doi:10.1038/nm.2757 · Cited by 210
Tat-Conjugated PAMAM Dendrimers as Delivery Agents for Antisense and siRNA Oligonucleotides
Kang, H. M., DeLong, R. K., Fisher, M. H., & Juliano, R. L. (2005) · Pharmaceutical Research · doi:10.1007/s11095-005-8330-5 · Cited by 197
Mäe, M. A., Andaloussi, S. E., Lundin, P., Oskolkov, N., Johansson, H. J., Guterstam, P., & Langel, Ü. (2008) · Journal of Controlled Release · doi:10.1016/j.jconrel.2008.11.025 · Cited by 192
“As a result of the association between alternative splicing and numerous pathological conditions, splice correction, together with other forms of gene regulation [4], is gaining increasing attention as a therapeutic target [3].”
BackgroundLönnberg, H. (2009) · Bioconjugate Chemistry · doi:10.1021/bc800406a · Cited by 188
Particle size affects the cellular response in macrophages
Yue, H., Wei, W., Yue, Z., Lv, P., Wang, L., Ma, G., & Su, Z. G. (2010) · European Journal of Pharmaceutical Sciences · doi:10.1016/j.ejps.2010.09.006 · Cited by 185
Distinct Uptake Routes of Cell-Penetrating Peptide Conjugates
Lundin, P., Johansson, H. J., Guterstam, P., Holm, T., Hansen, M., Langel, Ü., & Andaloussi, S. E. (2008) · Bioconjugate Chemistry · doi:10.1021/bc800212j · Cited by 192
Genetic medicines: treatment strategies for hereditary disorders
O’Connor, T. P., & Crystal, R. G. (2006) · Nature Reviews Genetics · doi:10.1038/nrg1829 · Cited by 188
“However, whereas newer generation ASO agents show increased stability, other crucial properties, including cellular uptake efficiencies, target specificity and binding affinities can be compromise”
BackgroundA Novel Cell-penetrating Peptide, M918, for Efficient Delivery of Proteins and Peptide Nucleic Acids
Andaloussi, S. E., Johansson, H. J., Holm, T., & Langel, Ü. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300255 · Cited by 173
“The main techniques to knock down the expression of proteins include the use of antisense oligonucleotides (asONs) and small interfering RNAs”
BackgroundAgarwala, P., Pandey, S., & Maiti, S. (2015) · Organic & Biomolecular Chemistry · doi:10.1039/c4ob02681k · Cited by 175
Kaur, H., Arora, A., Wengel, J., & Maiti, S. (2006) · Biochemistry · doi:10.1021/bi060307w · Cited by 168
Design, Synthesis, and Properties of 2‘,4‘-BNA NC : A Bridged Nucleic Acid Analogue
Rahman, S. M. A., Seki, S., Obika, S., Yoshikawa, H., Miyashita, K., & Imanishi, T. (2008) · Journal of the American Chemical Society · doi:10.1021/ja710342q · Cited by 164
Antisense Drug Discovery and Development
Yamamoto, T., Nakatani, M., Narukawa, K., & Obika, S. (2011) · Future Medicinal Chemistry · doi:10.4155/fmc.11.2 · Cited by 161
Lipid Conjugated Oligonucleotides: A Useful Strategy for Delivery
Raouane, M., Desmaële, D., Urbinati, G., Massaad-Massade, L., & Couvreur, P. (2012) · Bioconjugate Chemistry · doi:10.1021/bc200422w · Cited by 161
Niittymäki, T., & Lönnberg, H. (2005) · Organic & Biomolecular Chemistry · doi:10.1039/b509022a · Cited by 147
Marcucci, G., Stock, W., Dai, G., Klisovic, R. B., Liu, S., Klisovic, M. I., Blum, W. G., Kefauver, C. A., Sher, D. A., Green, M., et al. (2005) · Journal of Clinical Oncology · doi:10.1200/jco.2005.09.118 · Cited by 141
Gagnon, K. T., Pendergraff, H. M., Deleavey, G. F., Swayze, E. E., Potier, P., Randolph, J., Roesch, E. B., CHATTOPADHYAYA, J. B., Damha, M. J., Bennett, C. F., et al. (2010) · Biochemistry · doi:10.1021/bi101208k · Cited by 139
“are generally nontoxic when introduced into live animals at effective doses (69-71).”
Corradini, R., Sforza, S., Tedeschi, T., & Marchelli, R. (2007) · Chirality · doi:10.1002/chir.20372 · Cited by 139
Yan, H., Gu, W., Yang, J., Bi, V., Shen, Y., Lee, E. K., Winters, K. A., Komorowski, R., Zhang, C., Patel, J., et al. (2009) · Journal of Pharmacology and Experimental Therapeutics · doi:10.1124/jpet.108.147009 · Cited by 134
“The ASO approach, which has been widely used as a validation tool in preclinical studies, has also been applied to target GCGR and is in the early stages of clinical development (Crooke, 2004a,b; Liang et al., 2004; Sloop et al., 2004).”
Antisense inhibition of apolipoprotein (a) to lower plasma lipoprotein (a) levels in humans
Graham, M. J., Viney, N., Crooke, R. M., & Tsimikas, S. (2016) · Journal of Lipid Research · doi:10.1194/jlr.r052258 · Cited by 133
Identification and validation of cell surface antigens for antibody targeting in oncology
Carter, P. M., Smith, L. M., & Ryan, M. C. (2004) · Endocrine Related Cancer · doi:10.1677/erc.1.00766 · Cited by 130
“…alternative functional validation techniques that include RNA interference (RNAi) (Boese et al. 2003, Lavery & King 2003), antisense (Kurreck 2003, Crooke 2004), ribozymes (Castanotto et al. 2002, Kashani-Sabet 2002), gene knock outs, transgenic animals (Tornell & Snaith 2002), viral…”
BackgroundSelective Enhancement of Nucleases by Polyvalent DNA-Functionalized Gold Nanoparticles
Prigodich, A. E., Alhasan, A. H., & Mirkin, C. A. (2011) · Journal of the American Chemical Society · doi:10.1021/ja110833r · Cited by 127
van Deventer, S. J. H., Wedel, M. K., Baker, B. F., Xia, S., Chuang, E., & Miner, P. B. (2006) · Alimentary Pharmacology & Therapeutics · doi:10.1111/j.1365-2036.2006.02910.x · Cited by 127
Hung, G., Xiao, X., Peralta, R. Q., Bhattacharjee, G., Murray, S., Norris, D., Guo, S., & Monia, B. P. (2013) · Nucleic Acid Therapeutics · doi:10.1089/nat.2013.0443 · Cited by 126
“Although much has been learned about the basic mechanisms and the pharmacologic, pharmacokinetic, and toxicological properties of antisense molecules (Crooke, 2004), the exact mechanisms by which ASOs are taken up by cells, trafficked intracellularly, and cleared are still unclear.”
miRNAs: Key Players in Neurodegenerative Disorders and Epilepsy
Karnati, H. K., Panigrahi, M. K., Gutti, R. K., Greig, N. H., & Tamargo, I. A. (2015) · Journal of Alzheimer s Disease · doi:10.3233/jad-150395 · Cited by 126
Roh, Y. H., Lee, J. B., Shopsowitz, K. E., Dreaden, E. C., Morton, S. W., Poon, Z., Hong, J., Yamin, I., Bonner, D. K., & Hammond, P. T. (2014) · ACS Nano · doi:10.1021/nn502596b · Cited by 125
Intracellular delivery of an anionic antisense oligonucleotide via receptor-mediated endocytosis
Alam, M. R., Dixit, V., Kang, H. M., Li, Z., Chen, X. S., Trejo, J., Fisher, M. H., & Juliano, R. L. (2008) · Nucleic Acids Research · doi:10.1093/nar/gkn115 · Cited by 124
Antisense Oligonucleotide: Basic Concepts and Therapeutic Application in Inflammatory Bowel Disease
Fusco, D. D., Dinallo, V., Marafini, I., Figliuzzi, M. M., Romano, B., & Monteleone, G. (2019) · Frontiers in Pharmacology · doi:10.3389/fphar.2019.00305 · Cited by 126
“…not be designed into polymorphic/mutated regions of the genome and selection should exclude oligonucleotides targeting four contiguous guanosine residues in order to avoid generation of tetraplexes via Hoogsteen base-pair formation (Sen and Gilbert, 1992; Benimetskaya et al., 1997; Crooke, 2004).”
BackgroundCell-penetrating-peptide-based delivery of oligonucleotides: an overview
Abes, R., Arzumanov, A. A., Moulton, H. M., Abes, S., Ivanova, G. D., Iversen, P. L., Gait, M. J., & Lebleu, B. (2007) · Biochemical Society Transactions · doi:10.1042/bst0350775 · Cited by 122
Opportunities for yeast metabolic engineering: Lessons from synthetic biology
Krivoruchko, A., Siewers, V., & Nielsen, J. (2011) · Biotechnology Journal · doi:10.1002/biot.201000308 · Cited by 121
“Such hybridization events can result in steric hindrance, preventing translation, or facilitate the cleavage of their target by dsRNA cleaving enzymes [57].”
BackgroundBhindi, R., Fahmy, R. G., Lowe, H. C., Chesterman, C. N., Dass, C. R., Cairns, M. J., Saravolac, E. G., Sun, L., & Khachigian, L. M. (2007) · American Journal Of Pathology · doi:10.2353/ajpath.2007.070120 · Cited by 121
Frameworks for Programming Biological Function through RNA Parts and Devices
Win, M. N., Liang, J. C., & Smolke, C. D. (2009) · Chemistry & Biology · doi:10.1016/j.chembiol.2009.02.011 · Cited by 119
“Natural antisense RNAs have been characterized that actuate through a variety of mechanisms, including cleavage of antisense-target hybrids by dsRNA-cleaving enzymes and steric inhibition of gene expression machinery by the bound antisense (Crooke, 2004).”
BackgroundMicroRNAs as targets for antisense-based therapeutics
Stenvang, J., & Kauppinen, S. (2007) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.8.1.59 · Cited by 115
“However, extensive basic information on their pharmacokinetic properties and toxicity has been obtained [76,77] , which can now be applied to siRNA and especially to oligonucleotides targeting miRNAs and is therefore likely to pave the way for faster testing in clinical trials.”
BackgroundGromovsky, A. D., Schugar, R. C., Brown, A. L., Helsley, R. N., Burrows, A. C., Ferguson, D., Zhang, R., Sansbury, B. E., Lee, R. G., Morton, R. E., et al. (2018) · Arteriosclerosis, Thrombosis, and Vascular Biology · doi:10.1161/atvbaha.117.309660 · Cited by 115
Mansoor, M., & Melendez, A. J. (2008) · Gene Regulation and Systems Biology · doi:10.4137/grsb.s418 · Cited by 115
“Gene Regulation and Systems Biology 2008:2 In addition, there is the risk of ODNs being degraded to carcinogenic or mutagenic metabolites [60].”
BackgroundPharmacological strategies for lowering LDL cholesterol: statins and beyond
Brautbar, A., & Ballantyne, C. M. (2011) · Nature Reviews Cardiology · doi:10.1038/nrcardio.2011.2 · Cited by 115
“The ASO can inhibit mRNA translation and mRNA splicing, and eventually lead to its enzymatic degradation by ribonuclease”
BackgroundSmall molecule-mediated inhibition of translation by targeting a native RNA G-quadruplex
Bugaut, A., Rodriguez, R., Kumari, S., Hsu, S. D., & Balasubramanian, S. (2010) · Organic & Biomolecular Chemistry · doi:10.1039/c002418j · Cited by 114
Disease-modifying and symptomatic treatment of amyotrophic lateral sclerosis
Dorst, J., Ludolph, A. C., & Huebers, A. (2017) · Therapeutic Advances in Neurological Disorders · doi:10.1177/1756285617734734 · Cited by 111
Nucleic Acids as Therapeutic Agents
Álvarez-Salas, L. M. (2008) · Current Topics in Medicinal Chemistry · doi:10.2174/156802608786141133 · Cited by 105
Miner, P. B., Wedel, M. K., Xia, S., & Baker, B. F. (2006) · Alimentary Pharmacology & Therapeutics · doi:10.1111/j.1365-2036.2006.02837.x · Cited by 104
Novel Pharmacological Approaches to the Treatment of Type 2 Diabetes
Verspohl, E. J. (2012) · Pharmacological Reviews · doi:10.1124/pr.110.003319 · Cited by 103
“They are mostly 20-base chimeric oligonucleotides, the first and last five bases having a 2 -O-(2-methoxy)-ethyl modification (Crooke, 2004).”
BackgroundNew therapeutic principles in dyslipidaemia: focus on LDL and Lp(a) lowering drugs
Norata, G. D., Ballantyne, C. M., & Catapano, A. L. (2013) · European Heart Journal · doi:10.1093/eurheartj/eht088 · Cited by 99
“Following hybridization to the mRNA, the ASO inhibits translation and splicing, leading to mRNA degradation by RNase.(1) As ASO kinetics are characterized by a large and rapid distribution to the liver, this approach is quite attractive to reduce synthesis of proteins, such as apoB, in the liver(2) (Figure 1).”
BackgroundThe role of TGF-β in bone metastasis: novel therapeutic perspectives
Buijs, J. T., Stayrook, K. R., & Guise, T. A. (2012) · BoneKEy Reports · doi:10.1038/bonekey.2012.96 · Cited by 99
Yang, X., Koh, C. G., Liu, S., Pan, X., Santhanam, R., Yu, B., Peng, Y., Pang, J., Golan, S., Talmon, Y., et al. (2008) · Molecular Pharmaceutics · doi:10.1021/mp800149s · Cited by 97
Innovative nanotechnologies for the delivery of oligonucleotides and siRNA
Toub, N., Malvy, C., Fattal, E., & Couvreur, P. (2006) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2006.07.093 · Cited by 96
Aptamers Embedded in Polyacrylamide Nanoparticles: A Tool for in Vivo Metabolite Sensing
Nielsen, L. J., Olsen, L. F., & Özalp, V. C. (2010) · ACS Nano · doi:10.1021/nn100635j · Cited by 95
“AptaNP protection is an important criterion for in vivo utilization of nanosensors because of the otherwise rapid degradation of foreign oligonucleotides in blood and in cytoplasm as reported for in vivo studies.(13,14) Structural instability of aptamers under different buffer, ionic, and temperature conditions is ano…”
BackgroundMicroRNAs in parasitic diseases: Potential for diagnosis and targeting
Manzano-Román, R., & Siles‐Lucas, M. (2012) · Molecular and Biochemical Parasitology · doi:10.1016/j.molbiopara.2012.10.001 · Cited by 94
Genetic modification of stem cells for transplantation
Phillips, M. I., & Tang, Y. (2007) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2007.08.035 · Cited by 96
“[18] Antisense inhibition although widely used in research and approved for clinical treatment [19] is not perfect.”
MethodDeSouza, C. T., ARAUJO, E. P. D., Stoppiglia, L. F., Pauli, J. R., Ropelle, E. R., Rocco, S. A., Marin, R. M., Franchini, K. G., Carvalheira, J. B. C., Saad, M. J. A., et al. (2007) · The FASEB Journal · doi:10.1096/fj.06-7148com · Cited by 93
Niu, C., Prakash, T. P., Kim, A., Quach, J., Huryn, L. A., Yang, Y., Lopez, E., Jazayeri, A., Hung, G., Sopher, B. L., et al. (2018) · Science Translational Medicine · doi:10.1126/scitranslmed.aap8677 · Cited by 89
“This technique uses synthetic single strands of 15 to 25 nucleotides of perfect (or nearperfect) sequence complementary to bind to a tar get RNA and thereby produce a DNARNA heteroduplex that is a substrate for degradation by ribonuclease (RNase) H or will inter fere with RNA processing or translation (20).”
MethodThe Role of TGF-β in Bone Metastases
Trivedi, T., Pagnotti, G. M., Guise, T. A., & Mohammad, K. S. (2021) · Biomolecules · doi:10.3390/biom11111643 · Cited by 96
Overcoming biological barriers to in vivo efficacy of antisense oligonucleotides
White, P. J., Anastasopoulos, F., Pouton, C. W., & Boyd, B. J. (2009) · Expert Reviews in Molecular Medicine · doi:10.1017/s1462399409001021 · Cited by 87
“Proceedings of the National Academy of Sciences of the United States of America 102, 198-203 101 Crooke, R.M. et al. (2005) An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis. Journal of Lipid Research 46, 872-884 102 Guha, M. et al. (2007) Spe…”
BackgroundInfluentialCoagulation factor XI as a novel target for antithrombotic treatment
Löwenberg, E. C., Meijers, J. C. M., Monia, B. P., & Levi, M. M. (2010) · Journal of Thrombosis and Haemostasis · doi:10.1111/j.1538-7836.2010.04031.x · Cited by 85
Preparation of a Multitopic Glycopeptide−Oligonucleotide Conjugate
Singh, Y., Renaudet, O., Defrancq, É., & Dumy, P. (2005) · Organic Letters · doi:10.1021/ol050134n · Cited by 85
Controlled delivery of antisense oligonucleotides: a brief review of current strategies
Zhao, X., Pan, F., Holt, C. M., Lewis, A. L., & Lu, J. R. (2009) · Expert Opinion on Drug Delivery · doi:10.1517/17425240902992894 · Cited by 82
“Antisense oligonucleotides (ASOs) are short single-stranded molecules, each typically < 30 nucleotides in length [1-3].”
BackgroundBruno, I., Jin, W., & Cote, G. J. (2004) · Human Molecular Genetics · doi:10.1093/hmg/ddh272 · Cited by 84
Recent Developments in Peptide-Based Nucleic Acid Delivery
Veldhoen, S., Laufer, S. D., & Restle, T. (2008) · International Journal of Molecular Sciences · doi:10.3390/ijms9071276 · Cited by 88
“By far the most common use of oligonucleotides as inhibitors of gene expression is the so-called antisense approach (for a review see: [199, 200] ).”
BackgroundTranscriptome-Wide Off-Target Effects of Steric-Blocking Oligonucleotides
Holgersen, E. M., Gandhi, S., Zhou, Y., Kim, J., Vaz, B., Bogojeski, J. J., Bugno, M., Shalev, Z., Cheung-Ong, K., Gonçalves, J., et al. (2021) · Nucleic Acid Therapeutics · doi:10.1089/nat.2020.0921 · Cited by 81
“Oligonucleotides acting through this mechanism are known as gapmers, due to the presence of DNA bases flanked by modified bases (Crooke, 2004; X. Shen & Corey, 2018).”
Zhang, Y., Lai, L., Liu, Y., Chen, B., Yao, J., Zheng, P., Pan, Q., & Zhu, W. (2022) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.1c23808 · Cited by 81
Fischer, P. M. (2006) · Medicinal Research Reviews · doi:10.1002/med.20093 · Cited by 80
Mahato, R. I., Cheng, K., & Guntaka, R. V. (2005) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2.1.3 · Cited by 78
“Modulation of aberrant gene expression by antisense oligodeoxynucleotides (ODNs), triplex forming oligodeoxynucleotides (TFOs) and double-stranded small interfering RNAs (siRNAs) offer great promise for the treatment of many severe and debilitating diseases [1-7].”
BackgroundRecent Progress in Chemically Modified siRNAs
Gaglione, M., & Messere, A. (2010) · Mini-Reviews in Medicinal Chemistry · doi:10.2174/138955710791384036 · Cited by 76
“Three generations of chemically modified ASOs have been developed to enhance nuclease resistance, prolong tissue half-life, reduce off-target effects and increase affinity and potency [7, 17-24].”
BackgroundDinç, E., Ceppi, M. G., Tóth, S. Z., Bottka, S., & Schansker, G. (2012) · Biochimica et Biophysica Acta (BBA) - Bioenergetics · doi:10.1016/j.bbabio.2012.02.003 · Cited by 76
“In principle, they are able to interfere with each step of nucleic acid metabolism, preferentially with transcription, splicing and translation [35–39].”
BackgroundWang, S., Allen, N., Vickers, T. A., Revenko, A. S., Sun, H., Liang, X., & Crooke, S. T. (2018) · Nucleic Acids Research · doi:10.1093/nar/gky145 · Cited by 75
TGF-β in the Bone Microenvironment: Role in Breast Cancer Metastases
Buijs, J. T., Stayrook, K. R., & Guise, T. A. (2011) · Cancer Microenvironment · doi:10.1007/s12307-011-0075-6 · Cited by 76
Srivastava, P., Barman, J., Pathmasiri, W., Plashkevych, O., Wenska, M., & CHATTOPADHYAYA, J. B. (2007) · Journal of the American Chemical Society · doi:10.1021/ja071106y · Cited by 74
Role of TGF-β in breast cancer bone metastases
Chiechi, A., Waning, D. L., Stayrook, K. R., Buijs, J. T., Guise, T. A., & Mohammad, K. S. (2013) · Advances in Bioscience and Biotechnology · doi:10.4236/abb.2013.410a4003 · Cited by 76
“ASO’s inhibit mRNA function and protein synthesis via modulation of splicing and inhibition of translation [131,132].”
BackgroundIn Vivo Delivery Aspects of miRNA, shRNA and siRNA
Khatri, N., Rathi, M. N., Baradia, D., Trehan, S., & Misra, A. R. (2012) · Critical Reviews in Therapeutic Drug Carrier Systems · doi:10.1615/critrevtherdrugcarriersyst.v29.i6.20 · Cited by 69
Annexin A2 facilitates endocytic trafficking of antisense oligonucleotides
Wang, S., Sun, H., Tanowitz, M., Liang, X., & Crooke, S. T. (2016) · Nucleic Acids Research · doi:10.1093/nar/gkw595 · Cited by 72
Kiviniemi, A., Virta, P., & Lönnberg, H. (2008) · Bioconjugate Chemistry · doi:10.1021/bc800221p · Cited by 71
RNAi: a novel antisense technology and its therapeutic potential.
Dallas, A., & Vlassov, A. V. (2006) · PubMed · Cited by 71
Wang, S., Sun, H., Tanowitz, M., Liang, X., & Crooke, S. T. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx231 · Cited by 70
Meyer, M., & Wagner, E. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.1062 · Cited by 68
“Reviewing the extensive research in therapeutic knockdown of genes by naked antisense oligonucleotides or ribozymes goes beyond the scope of the current paper (for an in-depth discussion see Crooke, 2004; Patil et al ., 2005).”
Metadata
- Authors
- Crooke, S. T.
- Affiliations
- Ionis Pharmaceuticals (United States) (US)
- Corresponding author
- Crooke, Stanley T.
- ORCID
- Crooke, Stanley T.
- Venue
- Annual Review of Medicine
- Publisher
- Annual Reviews
- Published
- 2004-02-01
- Volume
- 55
- Issue
- 1
- Pages
- 61-95
- ISSN
- 0066-4219 (ISSN-L)
- 1545-326X
- OpenAlex
- W2138668121
- Cited by
- 417
- References
- 243
- Citations by year
- 2026: 1 · 2025: 5 · 2024: 5 · 2023: 10 · 2022: 7 · 2021: 8 · 2020: 3 · 2019: 9 · 2018: 5 · 2017: 15 · 2016: 12 · 2015: 14 · 2014: 16 · 2013: 24 · 2012: 32
- FWCI
- 3.07
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Annual Review of Medicine (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › DNA and Nucleic Acid Chemistry
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Research and Splicing
- Keywords
- antisense technology, oligonucleotide analogues, RNA hybridization, RNA degradation, RNA splicing, antisense drug development, pharmacokinetics, toxicity, target validation
- MeSH terms
- Animals, Humans, Oligonucleotides, Antisense, Biomedical Technology
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
