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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“Antisense oligodeoxyribonucleotides (ASOs, ODNs) are short, synthetic, single-stranded DNA molecules designed to specifically bind selected RNA or DNA sequences through Watson–Crick complementary base pairing [1,2].”
Advances in Therapeutic Applications of CRISPR Genome Editing for Spinal Pain Management
Kang, C. Y., Been, K. W., Kang, M. R., Choi, M. S., Kang, R. H., Hur, J. W., & Hur, J. K. (2025) · Neurospine · doi:10.14245/ns.2550462.231 · Cited by 15
Experimental Surgical Therapies for Huntington's Disease
Demeestere, J., & Vandenberghe, W. (2010) · CNS Neuroscience & Therapeutics · doi:10.1111/j.1755-5949.2010.00209.x · Cited by 11
“The siRNAs are then immediately available for unwinding, assembly with RISC and hybridization with the target mRNA.”
BackgroundInfluential
Inhibition of porcine reproductive and respiratory syndrome virus replication in vitro using DNA-based short antisense oligonucleotides
Zheng, L., Li, X. O., Zhu, L., Li, W., Bi, J., Yang, G., Yin, G., & Liu, J. (2015) · BMC Veterinary Research · doi:10.1186/s12917-015-0518-2 · Cited by 11
“viral infections, cancer growth, and inflammatory diseases [7–9].”
Background
Trafficking through the Rev/RRE Pathway Is Essential for Efficient Inhibition of Human Immunodeficiency Virus Type 1 by an Antisense RNA Derived from the Envelope Gene
Ward, A. M., Rekosh, D. M., & Hammarskjöld, M. (2008) · Journal of Virology · doi:10.1128/jvi.01520-08 · Cited by 11
“RNA (siRNA), and vector-delivered antisense RNA (11, 63).”
Background
Long-term Stable Cationic Solid Lipid Nanoparticles for the Enhanced Intracellular Delivery of SMAD3 Antisense Oligonucleotides in Activated Murine Macrophages
Jin, S., & Kim, C. (2012) · Journal of Pharmacy & Pharmaceutical Sciences · doi:10.18433/j3z312 · Cited by 11
“The ASOs have been investigated as a ‘magic bullet’ to treat several diseases since the 1970s (6, 7), which are designed to specifically hybridize to heterogeneous nuclear RNA or mature mRNA sequences for the inhibition of the expression of numerous genes.”
Background
Therapeutic Strategies to Target TGF-β in the Treatment of Bone Metastases
Buijs, J. T., Juárez, P., & Guise, T. A. (2011) · Current Pharmaceutical Biotechnology · doi:10.2174/138920111798808293 · Cited by 12
“RNase H cleaves the mRNA strand and leaves the ASO intact allowing it to continue to bind to other targets [118,119].”
Background
Adjuvanticity of a CTLA-4 3′ UTR complementary oligonucleotide for emulsion formulated recombinant subunit and inactivated vaccines
Li, X., Yang, L., Zhao, P., Yao, Y., Lu, F., Tu, L., Liu, J., Li, Z., Yu, Y., & Wang, L. (2017) · Vaccine · doi:10.1016/j.vaccine.2017.03.043 · Cited by 11
Reduction in intracellular HCV RNA and virus protein expression in human hepatoma cells following treatment with 2′-O-methyl-modified anti-core deoxyribozyme
Trépanier, J. B., Tanner, J. E., & Alfieri, C. (2008) · Virology · doi:10.1016/j.virol.2008.04.022 · Cited by 11
“These include their autocatalytic ability to cleave cytoplasmic RNAs without the requirement for RNAse H (Crooke, 2004).”
Background
Gab2 antisense oligonucleotide blocks rat basophilic leukemic cell functions
Chan, J. H. P., Liao, W., Lau, H. Y. A., & Wong, W. S. F. (2007) · International Immunopharmacology · doi:10.1016/j.intimp.2007.03.002 · Cited by 11
Imaging Gene Expression: Concepts and Future Outlook
Schipper, M. L., & Gambhir, S. S. (2006) · Medical radiology · doi:10.1007/3-540-30005-8_18 · Cited by 11
Frequency of C9orf72 and SOD1 mutations in 302 sporadic ALS patients from three German ALS centers
Yilmaz, R., Grehl, T., Eckrich, L., Marschalkowski, I., Weishaupt, K., Valkadinov, I., Simic, M., Brenner, D., Andersen, P. M., Wolf, J., et al. (2023) · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · doi:10.1080/21678421.2023.2165946 · Cited by 10
“This binding results in the degradation of the RNA or prevents its translation, leading to reduced protein expression (11).”
Background
PTPσ-mediated PI3P regulation modulates neurodegeneration in C9ORF72-ALS/FTD
Zhang, Z., Fu, X., Wright, N., Wang, W., Ye, Y., Asbury, J., Li, Y., Zhu, C., Wu, R., Wang, S., et al. (2025) · Neuron · doi:10.1016/j.neuron.2025.02.005 · Cited by 9
Reduction of gene expression by a hairpin-loop structured oligodeoxynucleotide: Alternative to siRNA and antisense
Kwok, T., Heinrich, J., Jung-Shiu, J., Meier, M. G., Mathur, S., & Moelling, K. (2009) · Biochimica et Biophysica Acta (BBA) - General Subjects · doi:10.1016/j.bbagen.2009.05.017 · Cited by 10
Molecular imaging and pharmacokinetics of 99mTc‐hTERT antisense oligonucleotide as a potential tumor imaging probe
Liu, M., Wang, R. F., Yan, P., Zhang, C. L., & Cui, Y. G. (2013) · Journal of Labelled Compounds and Radiopharmaceuticals · doi:10.1002/jlcr.3171 · Cited by 10
Hepatitis B virus: promising drug targets and therapeutic implications
Maepa, M. B., Bloom, K. M., Ely, A., & Arbuthnot, P. B. (2021) · Expert Opinion on Therapeutic Targets · doi:10.1080/14728222.2021.1915990 · Cited by 10
“Although NAPs are oligonucleotides, unlike ASOs they function in a sequence-independent manner.”
BackgroundInfluential
The application of antisense technology for crop improvement: A review
Tilahun, T., Bezie, Y., Kerisew, B., & Taye, M. (2021) · Cogent Food & Agriculture · doi:10.1080/23311932.2021.1910157 · Cited by 10
Suppression of invasion of a hamster pancreatic cancer cell line by antisense oligonucleotides mutation-matched to K-ras gene.
Morioka, C. Y., Machado, M. C. C., Saito, S., Nakada, Y., Matheus, A. S., Jukemura, J., Bacchella, T., Takahara, T., & Watanabe, A. (2005) · PubMed · Cited by 10
Synthesis of phosphorothioate oligonucleotide–peptide conjugates by solid phase fragment condensation
Diala, I., Osada, A., Maruoka, S., 今西高司, Murao, S., Ato, T., Ohba, H., & Fujii, M. (2007) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2007.09.101 · Cited by 10
Recent progress in oligonucleotide therapeutics: antisense to aptamers
Leaman, D. W. (2008) · Expert Opinion on Drug Discovery · doi:10.1517/17460441.3.9.997 · Cited by 10
“Such compounds have been used to target transcripts that encode proteins involved in the progression of nearly every main class of human disease, including cancers, cardiovascular diseases, inflammatory diseases, viral infections, respiratory diseases and many others [4-9] .”
BackgroundInfluential
Interfacial immobilisation of DNA molecules
Zhao, X., Pan, F., & Lu, J. R. (2007) · Annual Reports Section C (Physical Chemistry) · doi:10.1039/b605907b · Cited by 10
Polymer supported carbodiimide strategy for the synthesis of N-acylated derivatives of deoxy- and ribo purinenucleosides using active esters
Tripathi, S., Misra, K., & Sanghvi, Y. S. (2005) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2005.07.078 · Cited by 10
Stereodefined dinucleoside (3′,5′)-propionamidophosphonates and β-cyanoethylphosphonates and their incorporation into modified oligonucleotides
Woźniak, L. A., Bukowiecka‐Matusiak, M., Burzyńska-Pędziwiatr, I., & Stec, W. J. (2009) · Tetrahedron Letters · doi:10.1016/j.tetlet.2009.01.153 · Cited by 9
Modulation of Lipoprotein Metabolism by Antisense Technology: Preclinical Drug Discovery Methodology
Crooke, R. M., & Graham, M. J. (2013) · Methods in molecular biology · doi:10.1007/978-1-60327-369-5_14 · Cited by 9
Genetic modification of stem cells for cardiac, diabetic, and hemophilia transplantation therapies.
Phillips, M., & Tang, Y. (2012) · Progress in Molecular Biology and Translational Science · doi:10.1016/b978-0-12-398459-3.00013-7 · Cited by 8
On the Distant Horizon—Medical Therapy for Sensorineural Hearing Loss
Kelly, K. M., & Lalwani, A. K. (2015) · Otolaryngologic Clinics of North America · doi:10.1016/j.otc.2015.07.012 · Cited by 10
Direct and practical synthesis of 2′-O,4′-C-aminomethylene-bridged nucleic acid purine derivatives by transglycosylation
Umemoto, T., Masada, S., Miyata, K., Ogasawara-Shimizu, M., Murata, S., Nishi, K., Ogi, K., Hayase, Y., & Cho, N. (2017) · Tetrahedron · doi:10.1016/j.tet.2017.01.010 · Cited by 9
Oligonucleotide-Based Therapeutic Options against Hepatitis C Virus Infection
Trépanier, J. B., Tanner, J. E., & Alfieri, C. (2006) · Antiviral Therapy · doi:10.1177/135965350601100315 · Cited by 9
Protein kinase inhibitors as therapeutics in neurodegenerative and psychiatric disorders
Tridente, G., Jana, A., Nath, A., & Ashraf, G. M. (2023) · Receptor Tyrosine Kinases in Neurodegenerative and Psychiatric Disorders · doi:10.1016/b978-0-443-18677-6.00015-4 · Cited by 8
Advances in Antisense Oligo Technology for Sustainable Crop Protection
Patil, V., Jangra, S., & Ghosh, A. (2024) · Critical Reviews in Plant Sciences · doi:10.1080/07352689.2024.2394001 · Cited by 8
“When introduced into a biological system, ASOs selectively bind to their complementary RNA sequences (Crooke, 2004; Gagnon and Corey, 2019).”
MD simulation of LNA-modified human telomeric G-quadruplexes: a free energy calculation
Chaubey, A. K., Dubey, K. D., & Ojha, R. P. (2014) · Medicinal Chemistry Research · doi:10.1007/s00044-014-1182-y · Cited by 8
“In this context, the developments of nucleic acid with chemical modification rendering them high affinity and stability, since unmodified DNA or RNA oligonucleotides, have moderate affinities for complementary targets and low stability in biological fluids (Crooke, 2004).”
Background
Identification and Characterization of the V(D)J Recombination Activating Gene 1 in Long-Term Memory of Context Fear Conditioning
Castro‐Pérez, E., Soto-Soto, E., Pérez-Carambot, M., Dionisio-Santos, D. A., Saied‐Santiago, K., Ortiz‐Zuazaga, H., & de Ortı́z, S. P. (2015) · Neural Plasticity · doi:10.1155/2016/1752176 · Cited by 8
Synthesis and Stability of a 2′‐O‐[N‐(Aminoethyl)carbamoyl]methyladenosine‐Containing Dinucleotide
Milton, S., Ander, C., Honcharenko, D., Honcharenko, M., Yeheskiely, E., & Strömberg, R. (2013) · European Journal of Organic Chemistry · doi:10.1002/ejoc.201300699 · Cited by 8
Nanostructured polymer matrix for oligonucleotide separation
Zhang, J., Burger, C. P., & Chu, B. (2006) · Electrophoresis · doi:10.1002/elps.200500839 · Cited by 8
A two-step sulfurization for efficient solution-phase synthesis of phosphorothioate oligonucleotides
Ghisaidoobe, A. B. T., de Koning, M. C., Duynstee, H. I., Kortenaar, P. B. W. T., Overkleeft, H. S., Filippov, D. V., & van der MAREL, G. A. (2008) · Tetrahedron Letters · doi:10.1016/j.tetlet.2008.03.025 · Cited by 8
One-Pot Synthesis of TBMPS (bis[tert-Butyl)-1 Pyrenylmethyl-Silyl) Chloride as a Novel Fluorescent Silicon-Based Protecting Group for Protection of 5′-OH Nucleosides and Its Use as Purification Handle in Oligonucleotide Synthesis
Tripathi, S., Misra, K., & Sanghvi, Y. S. (2005) · Nucleosides Nucleotides & Nucleic Acids · doi:10.1080/15257770500230566 · Cited by 8
Oligonucleotide Formulations Prepared by High-Speed Electrospinning: Maximizing Loading and Exploring Downstream Processability
Hirsch, E., Nacsa, M., Pantea, E., Szabó, E., Vass, P., Domján, J., Farkas, A., Nyíri, Z., Eke, Z., Vígh, T., et al. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15030855 · Cited by 7
Antisense-Mediated Exon-Skipping to Induce Gene Knockdown
Disterer, P., & Khoo, B. C. E. (2012) · Methods in molecular biology · doi:10.1007/978-1-61779-767-5_19 · Cited by 7

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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