Cited by 417RNA Interference and Gene DeliveryDNA and Nucleic Acid ChemistryRNA Research and Splicing

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Progress in Antisense Technology

Crooke, Stanley T.·2004·Annual Review of Medicine

DOI 10.1146/annurev.med.55.091902.104408

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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.

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Background
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Scarozza, P., Schmitt, H., Monteleone, G., Neurath, M. F., & Atreya, R. N. (2019) · Frontiers in Pharmacology · doi:10.3389/fphar.2019.00314 · Cited by 36
“The second antisense mechanism is linked to the action of double-strand RNases which are activated by silence RNA (siRNA) molecules that are characterized by a double-strand oligonucleotide formed by a sense and an antisense RNA strand.”
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Therapeutic Potential of Mipomersen in the Management of Familial Hypercholesterolaemia
Gelsinger, C., Steinhagen‐Thiessen, E., & Kassner, U. (2012) · Drugs · doi:10.2165/11635060-000000000-00000 · Cited by 35
“Crooke RM, Graham MJ, Lemonidis KM, et al. An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemicmice without causing hepatic steatosis.”
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Multivalent DNA recognition by self-assembled clusters: deciphering structural effects by fragments screening and evaluation as siRNA vectors
Bartolami, E., Bessin, Y., Bettache, N., Gary‐Bobo, M., Garcia, M., Dumy, P., & Ulrich, S. (2015) · Organic & Biomolecular Chemistry · doi:10.1039/c5ob01404b · Cited by 39
Cellular Delivery and Biological Activity of Antisense Oligonucleotides Conjugated to a Targeted Protein Carrier
Kang, H. M., Alam, M. R., Dixit, V., Fisher, M. H., & Juliano, R. L. (2008) · Bioconjugate Chemistry · doi:10.1021/bc800270w · Cited by 35
“Antisense and siRNA oligonucleotides exhibit considerable promise as therapeutic agents (1, 2) While there are numerous examples of in vivo biological effects of oligonucleotides administered in free form, that is without use of a delivery system (3,4), some investigators have suggested that oligonucleotide-based ther…”
Background
Intravitreal Injection of Splice-switching Oligonucleotides to Manipulate Splicing in Retinal Cells
Gérard, X., Perrault, I., Münnich, A., Kaplan, J., & Rozet, J. (2015) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2015.24 · Cited by 34
Antisense Oligonucleotides, A Novel Developing Targeting Therapy
Karaki, S., Paris, C., & Rocchi, P. (2019) · IntechOpen eBooks · doi:10.5772/intechopen.82105 · Cited by 37
Antisense Recognition of the HER-2 mRNA: Effects of Phosphorothioate Substitution and Polyamines on DNA·RNA, RNA·RNA, and DNA·DNA Duplex Stability
Venkiteswaran, S., Vijayanathan, V., Shirahata, A., Thomas, T., & Thomas, T. (2004) · Biochemistry · doi:10.1021/bi0485272 · Cited by 34
Antisense apolipoprotein B therapy: where do we stand?
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Controlled intracellular localization and enhanced antisense effect of oligonucleotides by chemical conjugation
Kubo, T., Zhelev, Z., Bakalova, R., Ohba, H., Doi, K., & Fujii, M. (2005) · Organic & Biomolecular Chemistry · doi:10.1039/b507691a · Cited by 33
ASOptimizer: Optimizing antisense oligonucleotides through deep learning for IDO1 gene regulation
Hwang, G., Kwon, M., Seo, D., Kim, D. H., Lee, D., Lee, K., Kim, E., Kang, M., & Ryu, J. (2024) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2024.102186 · Cited by 32
DNA nanotechnology-based development of delivery systems for bioactive compounds
Mohri, K., Nishikawa, M., Takahashi, Y., & Takakura, Y. (2014) · European Journal of Pharmaceutical Sciences · doi:10.1016/j.ejps.2014.03.002 · Cited by 33
“…modified DNA analogs, including phosphorothioate (PS) ODN, 2-O-methyl ODN, morpholinos, methylphosphonate ODN, phosphoramidate ODN, and locked nucleic acid, have been developed for achieving enhanced stability against nucleases (Wagner, 1994; Crooke, 2004a, 2004b; Grünweller et al., 2003).”
Background
Piperine Modulates Protein Mediated Toxicity in Fragile X-Associated Tremor/Ataxia Syndrome through Interacting Expanded CGG Repeat (r(CGG)exp) RNA
Verma, A. K., Khan, E., Mishra, S. K., Jain, N., & Kumar, A. (2019) · ACS Chemical Neuroscience · doi:10.1021/acschemneuro.9b00282 · Cited by 32
Antisense therapy of MAdCAM-1 for trinitrobenzenesulfonic acid-induced murine colitis
Goto, A., Arimura, Y., Shinomura, Y., Imai, K., & Hinoda, Y. (2006) · Inflammatory Bowel Diseases · doi:10.1097/00054725-200608000-00013 · Cited by 32
“Antisense oligonucleotides (ASOs) remain a representative class of potential drugs for gene silencing in a sequence-specific manner.(23)”
BackgroundInfluential
Poly(propylacrylic acid) Enhances Cationic Lipid-Mediated Delivery of Antisense Oligonucleotides
Lee, L. K., Williams, C. L., Devore, D. I., & Roth, C. M. (2006) · Biomacromolecules · doi:10.1021/bm060114o · Cited by 32
4′-Alkoxy oligodeoxynucleotides: a novel class of RNA mimics
Liboska, R., Snášel, J., Barvı́k, I., Buděšı́nský, M., Pohl, R., Točı́k, Z., Páv, O., Rejman, D., Novák, P., & Rosenberg, I. (2011) · Organic & Biomolecular Chemistry · doi:10.1039/c1ob06148h · Cited by 32
Coxsackievirus-induced myocarditis: new trends in treatment
Liu, Z., Ji, Y., Yanagawa, B., Qiu, D., McManus, B. M., & Yang, D. (2005) · Expert Review of Anti-infective Therapy · doi:10.1586/14787210.3.4.641 · Cited by 32
“10 Aradottir E, Alonso EM, Shulman ST. Severe neonatal enteroviral hepatitis treated with pleconaril.”
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A New Strategy for Studying Protein Kinase B and Its Three Isoforms. Role of Protein Kinase B in Phosphorylating Glycogen Synthase Kinase-3, Tuberin, WNK1, and ATP Citrate Lyase
Sale, E. M., Hodgkinson, C. P., Jones, N. P., & Sale, G. J. (2005) · Biochemistry · doi:10.1021/bi050287i · Cited by 32
Alicaforsen for the treatment of inflammatory bowel disease
Jairath, V., Khanna, R., & Feagan, B. G. (2017) · Expert Opinion on Investigational Drugs · doi:10.1080/13543784.2017.1349753 · Cited by 31
Past, Present and Future of Therapeutic Interventions Targeting Leukocyte Trafficking in Inflammatory Bowel Disease
Panés, J., & Salas, A. (2018) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjy011 · Cited by 31
Antisense Oligonucleotides as Potential Therapeutics for Type 2 Diabetes
Chen, S., Sbuh, N., & Veedu, R. N. (2020) · Nucleic Acid Therapeutics · doi:10.1089/nat.2020.0891 · Cited by 30
Synthesis and Properties of a Bridged Nucleic Acid with a Perhydro-1,2-oxazin-3-one Ring
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Multitargeted Approach Using Antisense Oligonucleotides for the Treatment of Asthma
Allakhverdi, Z., ALLAM, M., GUIMOND, A., FERRARI, N., ZEMZOUMI, K., Seguin, R. M., Paquet, L., & RENZI, P. M. (2006) · Annals of the New York Academy of Sciences · doi:10.1196/annals.1348.047 · Cited by 31
Gene silencing approaches for the management of dyslipidaemia
Norata, G. D., Tibolla, G., & Catapano, A. L. (2013) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2013.01.010 · Cited by 30
Antisense inhibition of ICAM-1 expression as therapy provides insight into basic inflammatory pathways through early experiences in IBD
Philpott, J. R., & Miner, P. B. (2008) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.8.10.1627 · Cited by 30
Exon Skipping of Hepatic APOB Pre-mRNA With Splice-switching Oligonucleotides Reduces LDL Cholesterol In Vivo
Disterer, P., Al‐Shawi, R., Ellmerich, S., Waddington, S. N., Owen, J. S., Simons, J. P., & Khoo, B. C. E. (2013) · Molecular Therapy · doi:10.1038/mt.2012.264 · Cited by 30
“PS modification increases oligonucleotide stability to nucleases 23 but reduces binding affinity to RNA.”
Alternative Splicing: Therapeutic Target and Tool
García-Blanco, M. A. (2006) · Progress in molecular and subcellular biology · doi:10.1007/978-3-540-34449-0_3 · Cited by 30
Small‐Molecule‐Mediated Cleavage of RNA in Living Cells
Guan, L., & Disney, M. D. (2012) · Angewandte Chemie International Edition · doi:10.1002/anie.201206888 · Cited by 30
Chemical and Structural Implications of 1‘,2‘- versus 2‘,4‘- Conformational Constraints in the Sugar Moiety of Modified Thymine Nucleosides
Plashkevych, O., Chatterjee, S., Honcharenko, D., Pathmasiri, W., & CHATTOPADHYAYA, J. B. (2007) · The Journal of Organic Chemistry · doi:10.1021/jo070356u · Cited by 30
Current RNA strategies in treating cardiovascular diseases
Chia, S. P. S., Pang, J. K. S., & Soh, B.-S. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.01.028 · Cited by 32
“…induction, ASO hybridizes with targeted RNA via complementary Watson-Crick base pairing to form an ASO-RNA heteroduplex triggering RNA degradation mediated by RNase H. 39 Since RNase H is present in both nucleus and cytoplasm, ASOs ’ druggable targets could be extended to non-coding elements.”
Antisense Oligonucleotides and RNA Interference
Kher, G., Trehan, S., & Misra, A. R. (2010) · Elsevier eBooks · doi:10.1016/b978-0-12-384964-9.00007-4 · Cited by 30
“However, the favored design is to use chimeric molecules, such as a single ribonucleotide, which can be bound to its complementary oligonucleotide backbone, which then serves as a substrate for RNase H [36].”
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Transposable element-driven transcript diversification and its relevance to genetic disorders
Ayarpadikannan, S., Lee, H.-E., Han, K., & Kim, H. (2015) · Gene · doi:10.1016/j.gene.2015.01.039 · Cited by 32
Antisense oligonucleotides as therapeutics for hyperlipidaemias
Crooke, R. M. (2005) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.5.7.907 · Cited by 29
“The latter mechanisms occur when oligonucleotide analogue/ RNA duplexes become substrates for enzymatic degradation using various nucleases, including RNases H, double-stranded RNases or ribozymes [27-30].”
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Metabolism of GTI-2040, a phosphorothioate oligonucleotide antisense, using ion-pair reversed phase high performance liquid chromatography (HPLC) coupled with electrospray ion-trap mass spectrometry
Wei, X., Dai, G., Liu, Z., Cheng, H., Xie, Z., Marcucci, G., & Chan, K. K. (2006) · The AAPS Journal · doi:10.1208/aapsj080484 · Cited by 29
Effects of local mRNA structure on posttranscriptional gene silencing
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“PTGS is a natural phenomenon that can be accessed in the laboratory through the use of single-stranded antisense oligodeoxynucleotides (AONs), ribozymes, DNAzymes, or short interfering RNA (siRNA), all of which may direct degradation, or block translation, of the mRNA to which they are targeted (1, 2).”
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Nanostructure of Polyplexes Formed between Cationic Diblock Copolymer and Antisense Oligodeoxynucleotide and Its Influence on Cell Transfection Efficiency
Zhao, X., Pan, F., Zhang, Z., Grant, C. A., Ma, Y., Armes, S. P., Tang, Y., Lewis, A. L., Waigh, T. A., & Lu, J. R. (2007) · Biomacromolecules · doi:10.1021/bm7006482 · Cited by 29
Biological agents for ulcerative colitis: Hypes and hopes
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“Alicaforsen (ISIS 2302) is a 20 base phosphorothioate-modified antisense oligodeoxynucleotide able to hybridize to a sequence in the 3 0-untranslated region of the human ICAM-1 mRNA, causing the activation of the nuclease RNase-H with the cleavage of the heterodimer, resulting in a reduction of ICAM synthesis.(51)”
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Development of Apolipoprotein B Antisense Molecules as a Therapy for Hyperlipidemia
Thomas, T. A., & Ginsberg, H. N. (2009) · Current Atherosclerosis Reports · doi:10.1007/s11883-009-0078-7 · Cited by 27
“After binding to their complimentary RNA sequence, ASOs inhibit gene expression by several mechanisms, depending on the oligonucleotide modification [23].”
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Generation and characterization of regulatory dendritic cells derived from murine induced pluripotent stem cells
Zhang, Q., Fujino, M., Iwasaki, S., Hirano, H., Cai, S., Kitajima, Y., Xu, J., & Li, X. (2014) · Scientific Reports · doi:10.1038/srep03979 · Cited by 27
A GH receptor antisense oligonucleotide inhibits hepatic GH receptor expression, IGF-I production and body weight gain in normal mice
Tachas, G., Lofthouse, S. A., Wraight, C. J., Baker, B. F., Sioufi, N. B., Jarres, R., Berdeja, A., Rao, A., Kerr, L. M., D’Aniello, E., et al. (2006) · Journal of Endocrinology · doi:10.1677/joe.1.06553 · Cited by 27
Splicing therapeutics in SMN2 and APOB.
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RNA as a Versatile and Powerful Platform for Engineering Genetic Regulatory Tools
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“Antisense RNA molecules are single-stranded, trans-acting ncRNAs, whose sequences are complementary to target transcripts and usually consist of 12-20 complementary nucleotides (Crooke, 2004).”
Background
Effect of chemically modified IL-13 short interfering RNA on development of airway hyperresponsiveness in mice
Lively, T. N., Kossen, K., Balhorn, A., Koya, T., Zinnen, S. P., Takeda, K., Lucas, J. J., Polisky, B. A., Richards, I. M., & Gelfand, E. W. (2008) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2007.08.029 · Cited by 26
“[9] [10] [11] Moreover, recent publications demonstrate that chemically modified siRNAs can be used to avoid the dsRNA-induced immune response associated with some unmodified siRNA sequences.”
Background
2,2-Bis(ethoxycarbonyl)- and 2-(Alkylaminocarbonyl)-2-cyano-Substituted 3-(Pivaloyloxy)propyl Groups as Biodegradable Phosphate Protections of Oligonucleotides
Poijärvi-Virta, P., Heinonen, P., Virta, P., & Lönnberg, H. (2005) · Bioconjugate Chemistry · doi:10.1021/bc050143g · Cited by 26

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
DOI: 10.1146/annurev.med.55.091902.104408
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

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