Topoisomerase I inhibitors: camptothecins and beyond

Targeting the DNA repair defect in BRCA mutant cells as a therapeutic strategy

Farmer, H., McCabe, N., Lord, C. J., Tutt, A. N. J., Johnson, D. A., Richardson, T. B., Santarosa, M., Dillon, K. J., Hickson, I., Knights, C., et al. (2005) · Nature · doi:10.1038/nature03445 · Cited by 6,683

Multidrug resistance in cancer: role of ATP–dependent transporters

Gottesman, M. M., Fojo, T., & Bates, S. E. (2002) · Nature Reviews Cancer · doi:10.1038/nrc706 · Cited by 5,536

Targeting multidrug resistance in cancer

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Cell-cycle checkpoints and cancer

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Activation of the DNA damage checkpoint and genomic instability in human precancerous lesions

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Identification and characterization of a novel and specific inhibitor of the ataxia-telangiectasia mutated kinase ATM

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Genomic instability in laminopathy-based premature aging

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Mutation of TDP1, encoding a topoisomerase I–dependent DNA damage repair enzyme, in spinocerebellar ataxia with axonal neuropathy

Takashima, H., Boerkoel, C. F., John, J., Saifi, G. M., Salih, M. A. M., Armstrong, D., Mao, Y., Quiocho, F. A., Roa, B. B., Nakagawa, M., et al. (2002) · Nature Genetics · doi:10.1038/ng987 · Cited by 522

Role of p21 in Apoptosis and Senescence of Human Colon Cancer Cells Treated with Camptothecin

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S Phase and G2 Arrests Induced by Topoisomerase I Poisons Are Dependent on ATR Kinase Function

Cliby, W. A., Lewis, K. A., Lilly, K. K., & Kaufmann, S. H. (2002) · Journal of Biological Chemistry · doi:10.1074/jbc.m106287200 · Cited by 205

The multifaceted roles of PARP1 in DNA repair and chromatin remodelling

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Principles of cancer therapy: oncogene and non-oncogene addiction

Luo, J., Solimini, N. L., & Elledge, S. J. (2009) · Cell · doi:10.1016/j.cell.2009.02.024 · Cited by 1,765

γH2AX and cancer

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Alleviating Cancer Drug Toxicity by Inhibiting a Bacterial Enzyme

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Microsatellite Instability in Colorectal Cancer-the Stable Evidence

Vilar, E., & Gruber, S. B. (2010) · Nature Reviews Clinical Oncology · doi:10.1038/nrclinonc.2009.237 · Cited by 1,063

Exploiting senescence for the treatment of cancer

Wang, L., Lankhorst, L., & Bernards, R. (2022) · Nature Reviews Cancer · doi:10.1038/s41568-022-00450-9 · Cited by 867

Natural compounds for cancer treatment and prevention

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Topoisomerases as anticancer targets

Delgado, J. L., Hsieh, C.-M., Chan, N.-L., & Hiasa, H. (2018) · Biochemical Journal · doi:10.1042/bcj20160583 · Cited by 481

Topoisomerase inhibitors unsilence the dormant allele of Ube3a in neurons

Huang, H.-S., Allen, J. A., Mabb, A. M., King, I. F., Miriyala, J., Taylor-Blake, B., Sciaky, N., Dutton, J. W., Lee, H.-M., Chen, X., et al. (2012) · Nature · doi:10.1038/nature10726 · Cited by 387

Defective DNA Repair and Neurodegenerative Disease

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Chemotherapeutics and Radiation Stimulate MHC Class I Expression through Elevated Interferon-beta Signaling in Breast Cancer Cells

Wan, S., Pestka, S., Jubin, R. G., Lyu, Y. L., Tsai, Y.-C., & Liu, L. F. (2012) · PLoS ONE · doi:10.1371/journal.pone.0032542 · Cited by 295

SLFN11 Blocks Stressed Replication Forks Independently of ATR

Murai, J., Tang, S.-W., Leo, E., Baechler, S. A., Redon, C. E., Zhang, H., Al Abo, M., Rajapakse, V. N., Nakamura, E., Jenkins, L. M. M., et al. (2018) · Molecular Cell · doi:10.1016/j.molcel.2018.01.012 · Cited by 275

Irinotecan—Still an Important Player in Cancer Chemotherapy: A Comprehensive Overview

Kciuk, M., Marciniak, B., & Kontek, R. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21144919 · Cited by 266

Bacterial metabolism affects the C. elegans response to cancer chemotherapeutics

García-González, A. P., Ritter, A. D., Shrestha, S., Andersen, E. C., Yilmaz, L. S., & Walhout, A. J. M. (2017) · Cell · doi:10.1016/j.cell.2017.03.046 · Cited by 243

Phase I study of PARP inhibitor ABT-888 in combination with topotecan in adults with refractory solid tumors and lymphomas

Kummar, S., Chen, A., Ji, J., Zhang, Y., Reid, J. M., Ames, M., Jia, L., Weil, M., Speranza, G., Murgo, A. J., et al. (2011) · Cancer Research · doi:10.1158/0008-5472.can-11-1227 · Cited by 259

Payload diversification: a key step in the development of antibody–drug conjugates

Conilh, L., Sadilkova, L., Viricel, W., & Dumontet, C. (2023) · Journal of Hematology & Oncology · doi:10.1186/s13045-022-01397-y · Cited by 257

Coordination of DNA single strand break repair

Abbotts, R., & Wilson, D. M. (2017) · Free Radical Biology and Medicine · doi:10.1016/j.freeradbiomed.2016.11.039 · Cited by 240

Targeting hypoxia cell signaling for cancer therapy

Melillo, G. (2007) · Cancer and Metastasis Reviews · doi:10.1007/s10555-007-9059-x · Cited by 230

The intra-S phase checkpoint affects both DNA replication initiation and elongation: single-cell and -DNA fiber analyses

Seiler, J. A., Conti, C., Syed, A., Aladjem, M. I., & Pommier, Y. (2007) · Molecular and Cellular Biology · doi:10.1128/mcb.02278-06 · Cited by 226

Correlation between Ferumoxytol Uptake in Tumor Lesions by MRI and Response to Nanoliposomal Irinotecan in Patients with Advanced Solid Tumors: A Pilot Study

Ramanathan, R. K., Korn, R. L., Raghunand, N., Sachdev, J. C., Newbold, R. G., Jameson, G., Fetterly, G. J., Prey, J., Klinz, S. G., Kim, J., et al. (2017) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-16-1990 · Cited by 183

Increased antitumor activity of bevacizumab in combination with hypoxia inducible factor-1 inhibition

Rapisarda, A., Hollingshead, M., Uranchimeg, B., Bonomi, C. A., Borgel, S. D., Carter, J. P., Gehrs, B., Raffeld, M., Kinders, R. J., Parchment, R., et al. (2009) · Molecular Cancer Therapeutics · doi:10.1158/1535-7163.mct-09-0274 · Cited by 171

Novel Delivery of SN38 Markedly Inhibits Tumor Growth in Xenografts, Including a Camptothecin-11–Refractory Model

Sapra, P., Zhao, H., Mehlig, M., Malaby, J., Kraft, P., Longley, C., Greenberger, L. M., & Horak, I. D. (2008) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-07-4456 · Cited by 168

Optimal function of the DNA repair enzyme TDP1 requires its phosphorylation by ATM and/or DNA‐PK

Das, B. B., Antony, S., Gupta, S., Dexheimer, T. S., Redon, C. E., Garfield, S., Shiloh, Y., & Pommier, Y. (2009) · The EMBO Journal · doi:10.1038/emboj.2009.302 · Cited by 151

The MMS22L-TONSL Complex Mediates Recovery from Replication Stress and Homologous Recombination

O'Donnell, L., Panier, S., Wildenhain, J., Tkach, J. M., Al-Hakim, A., Landry, M.-C., Escribano-Diaz, C., Szilard, R. K., Young, J. T. F., Munro, M., et al. (2010) · Molecular Cell · doi:10.1016/j.molcel.2010.10.024 · Cited by 149

Alkylating agents and cancer therapy

Ralhan, R., & Kaur, J. (2007) · Expert Opinion on Therapeutic Patents · doi:10.1517/13543776.17.9.1061 · Cited by 147

Identification of the MMS22L-TONSL Complex that Promotes Homologous Recombination

Duro, E., Lundin, C., Ask, K., Sanchez-Pulido, L., MacArtney, T. J., Toth, R., Ponting, C. P., Groth, A., Helleday, T., & Rouse, J. (2010) · Molecular Cell · doi:10.1016/j.molcel.2010.10.023 · Cited by 125

Naphthoquinones: A continuing source for discovery of therapeutic antineoplastic agents

Qiu, H., Wang, P., Lin, H., Tang, C., Zhu, H., & Yang, Y. (2018) · Chemical Biology & Drug Design · doi:10.1111/cbdd.13141 · Cited by 118

From single-strand breaks to double-strand breaks during S-phase: a new mode of action of the Escherichia coli Cytolethal Distending Toxin: A new mode of action for CDT

Fedor, Y., Vignard, J., Nicolau-Travers, M.-L., Boutet-Robinet, E., Watrin, C., Salles, B., & Mirey, G. (2013) · Cellular Microbiology · doi:10.1111/cmi.12028 · Cited by 90

Old drug new use - Amoxapine and its metabolites as potent bacterial β-glucuronidase inhibitors for alleviating cancer drug toxicity

Kong, R., Liu, T., Zhu, X., Ahmad, S., Williams, A. L., Phan, A. T., Zhao, H., Scott, J. E., Yeh, L.-A., & Wong, S. T. C. (2014) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-14-0395 · Cited by 90

Combination Strategies to Improve Targeted Radionuclide Therapy

Chan, T. G., O’Neill, E., Habjan, C., & Cornelissen, B. (2020) · Journal of Nuclear Medicine · doi:10.2967/jnumed.120.248062 · Cited by 91

Tumor regression and curability of preclinical neuroblastoma models by PEGylated SN38 (EZN-2208), a novel topoisomerase I inhibitor

Pastorino, F., Loi, M., Sapra, P., Becherini, P., Cilli, M., Emionite, L., Ribatti, D., Greenberger, L. M., Horak, I. D., & Ponzoni, M. (2010) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-10-1354 · Cited by 79

Microcavity array (MCA)-based biosensor chip for functional drug screening of 3D tissue models

Kloß, D., Kurz, R., Jahnke, H.-G., Fischer, M., Rothermel, A., Anderegg, U., Simon, J. C., & Robitzki, A. A. (2008) · Biosensors and Bioelectronics · doi:10.1016/j.bios.2008.01.003 · Cited by 77

Identification of Circadian Determinants of Cancer Chronotherapy through In Vitro Chronopharmacology and Mathematical Modeling

Dulong, S., Ballesta, A., Okyar, A., & Lévi, F. (2015) · Molecular Cancer Therapeutics · doi:10.1158/1535-7163.mct-15-0129 · Cited by 68

Development of an apoptosis-assisted decellularization method for maximal preservation of nerve tissue structure

Cornelison, R. C., Wellman, S. M., Park, J. H., Porvasnik, S. L., Song, Y. H., Wachs, R. A., & Schmidt, C. E. (2018) · Acta Biomaterialia · doi:10.1016/j.actbio.2018.07.009 · Cited by 64

Repurposing of Cetuximab in antibody-directed chemotherapy-loaded nanoparticles in EGFR therapy-resistant pancreatic tumours

McDaid, W. J., Greene, M. K., Johnston, M. C., Pollheimer, E., Smyth, P., McLaughlin, K., Van Schaeybroeck, S., Straubinger, R. M., Longley, D. B., & Scott, C. J. (2019) · Nanoscale · doi:10.1039/c9nr07257h · Cited by 58

Synergistic Leukemia Eradication by Combined Treatment with Retinoic Acid and HIF Inhibition by EZN-2208 (PEG-SN38) in Preclinical Models of PML-RARα and PLZF-RARα–Driven Leukemia

Coltella, N., Valsecchi, R., Ponente, M., Ponzoni, M., & Bernardi, R. (2015) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-14-3022 · Cited by 49

Topotecan is a potent inhibitor of SUMOylation in glioblastoma multiforme and alters both cellular replication and metabolic programming

Bernstock, J. D., Ye, D., Gessler, F. A., Lee, Y.-J., Peruzzotti-Jametti, L., Baumgarten, P., Johnson, K. R., Maric, D., Yang, W., Kögel, D., et al. (2017) · Scientific Reports · doi:10.1038/s41598-017-07631-9 · Cited by 46

Potential Repurposing of Known Drugs as Potent Bacterial β-Glucuronidase Inhibitors

Ahmad, S., Hughes, M. A., Yeh, L.-A., & Scott, J. E. (2012) · SLAS Discovery · doi:10.1177/1087057112444927 · Cited by 44

Targeting the human gut microbiome with small-molecule inhibitors

Woo, A. Y. M., Aguilar Ramos, M. A., Narayan, R., Richards-Corke, K. C., Wang, M. L., Sandoval-Espinola, W. J., & Balskus, E. P. (2023) · Nature Reviews Chemistry · doi:10.1038/s41570-023-00471-4 · Cited by 42

In Vivo Topoisomerase I Inhibition Attenuates the Expression of Hypoxia-Inducible Factor 1α Target Genes and Decreases Tumor Angiogenesis

Guérin, E., Raffelsberger, W., Pencreach, E., Maier, A., Neuville, A., Schneider, A., Bachellier, P., Rohr, S., Petitprez, A., Poch, O., et al. (2012) · Molecular Medicine · doi:10.2119/molmed.2011.00120 · Cited by 44

Poly (ADP-ribose) polymerase 1 and neurodegenerative diseases: past, present, and future

Hu, M.-L., Pan, Y.-R., Yong, Y.-Y., Liu, Y., Yu, L., Qin, D.-L., Qiao, G., Law, B. Y.-K., Wu, J.-M., Zhou, X.-G., et al. (2023) · Ageing Research Reviews · doi:10.1016/j.arr.2023.102078 · Cited by 44

Iron Chelators with Topoisomerase-Inhibitory Activity and Their Anticancer Applications

Rao, V. A. (2013) · Antioxidants & Redox Signaling · doi:10.1089/ars.2012.4877 · Cited by 37

Clinico-genomic Characterization of ATM and HRD in Pancreas Cancer: Application for Practice

Park, W., O'Connor, C. A., Bandlamudi, C., Forman, D., Chou, J. F., Umeda, S., Reyngold, M., Varghese, A. M., Keane, F., Balogun, F., et al. (2022) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-22-1483 · Cited by 34

Development of novel apoptosis-assisted lung tissue decellularization methods

Song, Y. H., Maynes, M. A., Hlavac, N., Visosevic, D., Daramola, K. O., Porvasnik, S. L., & Schmidt, C. E. (2021) · Biomaterials Science · doi:10.1039/d1bm00032b · Cited by 33

A High Throughput Assay for Discovery of Bacterial β-Glucuronidase Inhibitors

Ahmad, S., Hughes, M. A., Lane, K. T., Redinbo, M. R., Yeh, L.-A., & Scott, J. E. (2011) · Current Chemical Genomics · doi:10.2174/1875397301105010013 · Cited by 29

The multifunctional protein PACS-1 is required for HDAC2- and HDAC3-dependent chromatin maturation and genomic stability

Mani, C., Tripathi, K., Luan, S., Clark, D. W., Andrews, J. F., Vindigni, A., Thomas, G., & Palle, K. (2020) · Oncogene · doi:10.1038/s41388-020-1167-x · Cited by 27

Y-box binding protein 1 enhances DNA topoisomerase 1 activity and sensitivity to camptothecin via direct interaction

Wu, Y., Wang, K.-Y., Li, Z., Liu, Y.-P., Izumi, H., Uramoto, H., Nakayama, Y., Ito, K.-I., & Kohno, K. (2014) · Journal of Experimental & Clinical Cancer Research · doi:10.1186/s13046-014-0112-7 · Cited by 17

Enhancing radiotherapy techniques for Triple-Negative breast cancer treatment

Sarlak, S., Pagès, G., & Luciano, F. (2025) · Cancer Treatment Reviews · doi:10.1016/j.ctrv.2025.102939 · Cited by 19

Tirapazamine Sensitizes Hepatocellular Carcinoma Cells to Topoisomerase I Inhibitors via Cooperative Modulation of Hypoxia-Inducible Factor-1α

Cai, T.-Y., Liu, X.-W., Zhu, H., Cao, J., Zhang, J., Ding, L., Lou, J.-S., He, Q.-J., & Yang, B. (2014) · Molecular Cancer Therapeutics · doi:10.1158/1535-7163.mct-13-0490 · Cited by 19

The evolving landscape of antibody-drug conjugates (ADCs) for treatment of prostate cancer

Lin, C., Hadfield, M. J., Santopietro, A., Lagos, G., Cheng, L., El-Deiry, W. S., Aggarwal, R., Mega, A., Hyams, E., & Carneiro, B. A. (2025) · npj Precision Oncology · doi:10.1038/s41698-025-01131-0 · Cited by 20

A High-Content Screen for C/EBPα Expression Identifies Novel Therapeutic Agents in Dedifferentiated Liposarcoma

Angeles, C. V., Velez, A., Rios, J., Laxa, B., Shum, D., Ruiz, P. D., Shen, Y., Ostrovnaya, I., Gularte-Mérida, R., Nacev, B. A., et al. (2022) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-19-2486 · Cited by 13

TDP1 splice-site mutation causes HAP1 cell hypersensitivity to topoisomerase I inhibition

Goh, C. G., Bader, A. S., Tran, T.-A., Belotserkovskaya, R., D’Alessandro, G., & Jackson, S. P. (2025) · Nucleic Acids Research · doi:10.1093/nar/gkae1163 · Cited by 5

AAV-mediated genome editing is influenced by the formation of R-loops

Puzzo, F., Crossley, M. P., Goswami, A., Zhang, F., Pekrun, K., Garzon, J. L., Cimprich, K. A., & Kay, M. A. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.09.035 · Cited by 4