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Gefitinib in Combination With Paclitaxel and Carboplatin in Advanced Non–Small-Cell Lung Cancer: A Phase III Trial—INTACT 2

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Oncogenically active MYD88 mutations in human lymphoma

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Dominant-negative mutations in the DNA-binding domain of STAT3 cause hyper-IgE syndrome

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Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial

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Enhanced DNA-Binding Activity of a Stat3-Related Protein in Cells Transformed by the Src Oncoprotein

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Effect of crizotinib on overall survival in patients with advanced non-small-cell lung cancer harbouring ALK gene rearrangement: a retrospective analysis

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Oncogene Addiction

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SOCS-1, a negative regulator of the JAK/STAT pathway, is silenced by methylation in human hepatocellular carcinoma and shows growth-suppression activity

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Mitochondrial STAT3 Supports Ras-Dependent Oncogenic Transformation

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The Ups and Downs of STAT Inhibition in Acute Myeloid Leukemia

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KRAS-Mutant Lung Cancer: Targeting Molecular and Immunologic Pathways, Therapeutic Advantages and Restrictions

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Clinical applications of oligonucleotides for cancer therapy

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RNA Therapeutics: Delivery Problems and Solutions—A Review

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Targeting STAT3 with Proteolysis Targeting Chimeras and Next-Generation Antisense Oligonucleotides

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B Cell Lymphoma Immunotherapy Using TLR9-Targeted Oligonucleotide STAT3 Inhibitors

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RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors

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A Phase 1b Study to Evaluate the Safety and Efficacy of Durvalumab in Combination With Tremelimumab or Danvatirsen in Patients With Relapsed or Refractory Diffuse Large B-Cell Lymphoma

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Altered pathways and targeted therapy in double hit lymphoma

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Simultaneous inhibition of endocytic recycling and lysosomal fusion sensitizes cells and tissues to oligonucleotide therapeutics

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Stat3 regulates desmoglein 3 transcription in epithelial keratinocytes

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An update on investigational therapies that target STAT3 for the treatment of cancer

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Discovery of 2-Amino-3-cyanothiophene Derivatives as Potent STAT3 Inhibitors for the Treatment of Osteosarcoma Growth and Metastasis

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Mechanisms and therapeutic prospect of the JAK-STAT signaling pathway in liver cancer

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The notorious R.N.A. in the spotlight - drug or target for the treatment of disease

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Considering the potential for gene-based therapy in prostate cancer

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Delivery of GalNAc-Conjugated Splice-Switching ASOs to Non-hepatic Cells through Ectopic Expression of Asialoglycoprotein Receptor

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Function of the Long Noncoding RNAs in Hepatocellular Carcinoma: Classification, Molecular Mechanisms, and Significant Therapeutic Potentials

Khan, A., & Zhang, X. (2022) · Bioengineering · doi:10.3390/bioengineering9080406 · Cited by 29

Stat3 inhibitor abrogates the expression of PD-1 ligands on lymphoma cell lines

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Oligonucleotide-based pharmaceuticals: Non-clinical and clinical safety signals and non-clinical testing strategies

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Silencing of STE20‐type kinase MST3 in mice with antisense oligonucleotide treatment ameliorates diet‐induced nonalcoholic fatty liver disease

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The long non-coding RNA SAMMSON is essential for uveal melanoma cell survival

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ERK5 Signalling and Resistance to ERK1/2 Pathway Therapeutics: The Path Less Travelled?

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Igniting Hope for Tumor Immunotherapy: Promoting the “Hot and Cold” Tumor Transition

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Targeted depletion of PIK3R2 induces regression of lung squamous cell carcinoma

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Targeting the Chromosomal Passenger Complex Subunit INCENP Induces Polyploidization, Apoptosis, and Senescence in Neuroblastoma

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CHOP-ASO Ameliorates Glomerular and Tubular Damage on Top of ACE Inhibition in Diabetic Kidney Disease

Shahzad, K., Fatima, S., Al‐Dabet, M. M., Gadi, I., Khawaja, H., Ambreen, S., Elwakiel, A., Klöting, N., Blüher, M., Nawroth, P. P., et al. (2021) · Journal of the American Society of Nephrology · doi:10.1681/asn.2021040431 · Cited by 27

Ailanthone suppresses the activity of human colorectal cancer cells through the STAT3 signaling pathway

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Transcription Factor Sp1 in the Expression of Genes Encoding Components of Mapk, JAK/STAT, and PI3K/Akt Signaling Pathways

Ivanenko, K. A., Prassolov, V., & Khabusheva, E. (2022) · Molecular Biology · doi:10.1134/s0026893322050089 · Cited by 29

The prognostic value and therapeutic targeting of myeloid-derived suppressor cells in hematological cancers

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The Receptor for Advanced Glycation Endproducts (RAGE) and Its Ligands S100A8/A9 and High Mobility Group Box Protein 1 (HMGB1) Are Key Regulators of Myeloid-Derived Suppressor Cells

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Novel Therapies in APOL1-Mediated Kidney Disease: From Molecular Pathways to Therapeutic Options

Vasquez‐Rios, G., de Cos, M., & Campbell, K. N. (2023) · Kidney International Reports · doi:10.1016/j.ekir.2023.08.028 · Cited by 27

Immunomodulation of hematological malignancies using oligonucleotides based-nanomedicines

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A STAT3–STING–IFN axis controls the metastatic spread of small cell lung cancer

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Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease

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MXD3 antisense oligonucleotide with superparamagnetic iron oxide nanoparticles: A new targeted approach for neuroblastoma

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Current advancements and future perspectives of long noncoding RNAs in lipid metabolism and signaling

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Progranulin sustains STAT 3 hyper‐activation and oncogenic function in colorectal cancer cells

Laudisi, F., Cherubini, F., Grazia, A. D., Dinallo, V., Fusco, D. D., Franzè, E., Ortenzi, A., Salvatori, I., Scaricamazza, S., Monteleone, I., et al. (2019) · Molecular Oncology · doi:10.1002/1878-0261.12552 · Cited by 26

Chronic lymphoproliferative disorder of NK‐cells: A single‐institution review with emphasis on relative utility of multimodality diagnostic tools

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Exploring Novel Frontiers: Leveraging STAT3 Signaling for Advanced Cancer Therapeutics

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Emerging Progress of RNA-Based Antitumor Therapeutics

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STAT3 axis in cancer and cancer stem cells: From oncogenesis to targeted therapies

Godugu, D., Chilamakuri, R., & Agarwal, S. (2025) · Biochimica et Biophysica Acta (BBA) - Reviews on Cancer · doi:10.1016/j.bbcan.2025.189461 · Cited by 26

STAT3 Inhibitors in Cancer: A Comprehensive Update

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Molecular Subtyping in Diffuse Large B Cell Lymphoma: Closer to an Approach of Precision Therapy

Karmali, R., & Gordon, L. I. (2017) · Current Treatment Options in Oncology · doi:10.1007/s11864-017-0449-1 · Cited by 24

The Non-Coding Transcriptome of Prostate Cancer: Implications for Clinical Practice

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CYTL1 inhibits tumor metastasis with decreasing STAT3 phosphorylation

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Nanomedicine for the Delivery of RNA in Cancer

Ghidini, M., Silva, S. G., Evangelista, J., Vale, M. L. C., Farooqı, A. A., & Pinheiro, M. (2022) · Cancers · doi:10.3390/cancers14112677 · Cited by 23

Oligonucleotide-Based Therapeutics for STAT3 Targeting in Cancer—Drug Carriers Matter

Molenda, S., Sikorska, A., Florczak, A., Lorenc, P., & Dams‐Kozlowska, H. (2023) · Cancers · doi:10.3390/cancers15235647 · Cited by 23

Tropisetron ameliorates cyclophosphamide-induced hemorrhagic cystitis in rats

Zirak, M. R., Karimi, G., Rahimian, R., Jafarian, A. H., Hayes, A. W., & Mehri, S. (2020) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2020.173310 · Cited by 22

Research progress on non-protein-targeted drugs for cancer therapy

Zhang, Y., Lu, L., Song, F., Zou, X., Liu, Y., Zheng, X., Qian, J., Gu, C., Huang, P. C., & Yang, Y. (2023) · Journal of Experimental & Clinical Cancer Research · doi:10.1186/s13046-023-02635-y · Cited by 22

Phosphotyrosine isosteres: past, present and future

Cerulli, R. A., & Kritzer, J. A. (2019) · Organic & Biomolecular Chemistry · doi:10.1039/c9ob01998g · Cited by 21

Potent antitumor effects of cell-penetrating peptides targeting STAT3 axis

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FAM72, Glioblastoma Multiforme (GBM) and Beyond

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Specific targeting of the KRAS mutational landscape in myeloma as a tool to unveil the elicited antitumor activity

Sacco, A., Federico, C., Todoerti, K., Ziccheddu, B., Palermo, V., Giacomini, A., Ravelli, C., Maccarinelli, F., Bianchi, G., Belotti, A., et al. (2021) · Blood · doi:10.1182/blood.2020010572 · Cited by 20

Future directions for medicinal chemistry in the field of oligonucleotide therapeutics

Hall, J. (2023) · RNA · doi:10.1261/rna.079511.122 · Cited by 21

Current Status and Future Direction of Immunotherapy in Urothelial Carcinoma

Lattanzi, M. W., & Balar, A. V. (2019) · Current Oncology Reports · doi:10.1007/s11912-019-0775-5 · Cited by 20

Knowing the tumour microenvironment to optimise immunotherapy

Merlano, M. C., Abbona, A., Denaro, N., & Garrone, O. (2019) · Acta Otorhinolaryngologica Italica · doi:10.14639/0392-100x-2481 · Cited by 19

Anwulignan is a novel JAK1 inhibitor that suppresses non‐small cell lung cancer growth

Xie, X., Wang, X., Shi, X., Zhang, Y., Laster, K. V., Liu, K., Dong, Z., & Kim, D. J. (2021) · Journal of Cellular and Molecular Medicine · doi:10.1111/jcmm.16289 · Cited by 20

Targeting Intercellular Communication in the Bone Microenvironment to Prevent Disseminated Tumor Cell Escape from Dormancy and Bone Metastatic Tumor Growth

Kreps, L. M., & Addison, C. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22062911 · Cited by 22

Targeting STAT3 in tumor-associated antigen-presenting cells as a strategy for kidney and bladder cancer immunotherapy

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Oncogenic STAT Transcription Factors as Targets for Cancer Therapy: Innovative Strategies and Clinical Translation

Wang, W., McDonald, M. C. L., Hariprasad, R., Hamilton, T., & Frank, D. A. (2024) · Cancers · doi:10.3390/cancers16071387 · Cited by 21

Autologous reconstitution of human cancer and immune system in vivo

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Trichothecin Inhibits Cancer-Related Features in Colorectal Cancer Development by Targeting STAT3

Qi, X., Li, M., Zhang, X., Dai, X.-F., Cui, J. T., Li, D., Gu, Q., Lv, Z., & Li, J. (2020) · Molecules · doi:10.3390/molecules25102306 · Cited by 21

Polarization of M2 Tumor-Associated Macrophages (TAMs) in Cancer Immunotherapy

Bui, I., & Bonavida, B. (2024) · Critical Reviews™ in Oncogenesis · doi:10.1615/critrevoncog.2024053830 · Cited by 18

Non-Viral RNA Therapies for Non-Small Cell Lung Cancer and Their Corresponding Clinical Trials

Tiwade, P. B., Fung, V., VanKeulen‐Miller, R., Narasipura, E. A., Ma, Y., & Fenton, O. S. (2025) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.4c00871 · Cited by 18

Precision cytokine modulation to overcome tumor microenvironment-driven resistance to immune checkpoint blockade

Wang, X., Wang, J., Wang, Q., Ding, G., Huang, Y., & Feng, Y. (2026) · Biochimica et Biophysica Acta (BBA) - Reviews on Cancer · doi:10.1016/j.bbcan.2026.189536 · Cited by 19

Selective targeting of MYC mRNA by stabilized antisense oligonucleotides

Gill, T., Wang, H., Bandaru, R., Lawlor, M. A., Lu, C., Nieman, L. T., Tao, J., Zhang, Y., Anderson, D. G., Ting, D. T., et al. (2021) · Oncogene · doi:10.1038/s41388-021-02053-4 · Cited by 21

Polymeric Carriers for Delivery of RNA Cancer Therapeutics

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Role of Alternative Splicing in Prostate Cancer Aggressiveness and Drug Resistance in African Americans

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Recent Advances in Medicinal Chemistry of Antisense Oligonucleotides

Ito, K. R., & Obika, S. (2016) · Elsevier eBooks · doi:10.1016/b978-0-12-409547-2.12420-5 · Cited by 19

STAT3 induces G9a to exacerbate HER3 expression for the survival of epidermal growth factor receptor-tyrosine kinase inhibitors in lung cancers

Chang, Y.-F., Lim, K., Chiang, Y.-W., Sie, Z., Chang, J., Ho, A., & Cheng, C. (2019) · BMC Cancer · doi:10.1186/s12885-019-6217-9 · Cited by 17

Introduction and History of the Chemistry of Nucleic Acids Therapeutics

Gait, M. J., & Agrawal, S. (2022) · Methods in molecular biology · doi:10.1007/978-1-0716-2010-6_1 · Cited by 18

The Small RNA Landscape in NSCLC: Current Therapeutic Applications and Progresses

Ciccone, G., Ibba, M. L., Coppola, G., Catuogno, S., & Esposito, C. L. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24076121 · Cited by 17

Reprogramming lymphocytes for the treatment of melanoma: From biology to therapy

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Chemical Modifications in RNA Interference and CRISPR/Cas Genome Editing Reagents

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Constant Activation of STAT3 Contributes to the Development of Adenomyosis in Females

Hiraoka, T., Hirota, Y., Aikawa, S., Iida, R., Ishizawa, C., Kaku, T., Hirata, T., Fukui, Y., Akaeda, S., Matsuo, M., et al. (2022) · Endocrinology · doi:10.1210/endocr/bqac044 · Cited by 17

Targeting STAT3 and STAT5 in Cancer

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Defective Ribonucleoproteins, Mistakes in RNA Processing, and Diseases

Mihailovic, M. K., Chen, A., Gonzalez‐Rivera, J. C., & Contreras, L. M. (2017) · Biochemistry · doi:10.1021/acs.biochem.6b01134 · Cited by 15

Oncosuppressors and Oncogenes: Role in Haemangioma Genesis and Potential for Therapeutic Targeting

Mabeta, P. (2018) · International Journal of Molecular Sciences · doi:10.3390/ijms19041192 · Cited by 16

The Medicinal Chemistry of RNase H-activating Antisense Oligonucleotides

Seth, P. P., & Swayze, E. E. (2019) · Royal Society of Chemistry eBooks · doi:10.1039/9781788015714-00032 · Cited by 16

Interferon regulatory factor 4 as a therapeutic target in adult T-cell leukemia lymphoma

Rauch, D. A., Olson, S. L., Harding, J. C., Sundaramoorthi, H., Kim, Y., Zhou, T., MacLeod, A. R., Challen, G. A., & Ratner, L. (2020) · Retrovirology · doi:10.1186/s12977-020-00535-z · Cited by 17

Redox responsive Pluronic micelle mediated delivery of functional siRNA: a modular nano-assembly for targeted delivery

Kadekar, S., Nawale, G. N., Rangasami, V. K., Joncour, V. L., Laakkonen, P. M., Hilborn, J. G., Varghese, O. P., & Oommen, O. P. (2021) · Biomaterials Science · doi:10.1039/d1bm00428j · Cited by 16

PEG-modification on the endo-position of an antisense oligonucleotide increases tumor accumulation via the EPR effect

Hagiwara, K., Kurihara, K., Honma, M., Yamamoto, J., & Shinohara, F. (2018) · Journal of Biomaterials Science Polymer Edition · doi:10.1080/09205063.2017.1422853 · Cited by 16

Cytokines and Janus kinase/signal transducer and activator of transcription signaling in prostate cancer: overview and therapeutic opportunities

Canesin, G., Krzyzanowska, A., Hellsten, R., & Bjartell, A. S. (2020) · Current Opinion in Endocrine and Metabolic Research · doi:10.1016/j.coemr.2020.02.004 · Cited by 16

DDIAS, DNA damage-induced apoptosis suppressor, is a potential therapeutic target in cancer

Im, J., Kang, M., Kim, B., & Won, M. (2023) · Experimental & Molecular Medicine · doi:10.1038/s12276-023-00974-6 · Cited by 14

Nucleic acid therapeutics as differentiation agents for myeloid leukemias

Kovecses, O., Mercier, F., & McKeague, M. (2024) · Leukemia · doi:10.1038/s41375-024-02191-0 · Cited by 18

STAT3 is constitutively acetylated on lysine 685 residues in chronic lymphocytic leukemia cells

Rozovski, U., Harris, D., Li, P., Liu, Z., Jain, P., Ferrajoli, A., Burger, J. A., Thompson, P. A., Jain, N., Wierda, W. G., et al. (2018) · Oncotarget · doi:10.18632/oncotarget.26110 · Cited by 17

Network potential identifies therapeutic miRNA cocktails in Ewing sarcoma

Weaver, D. T., Pishas, K. I., Williamson, D. F. K., Scarborough, J. A., Lessnick, S. L., Dhawan, A., & Scott, J. G. (2021) · PLoS Computational Biology · doi:10.1371/journal.pcbi.1008755 · Cited by 16

Preclinical study and parallel phase II trial evaluating antisense STAT3 oligonucleotide and checkpoint blockade for advanced pancreatic, non-small cell lung cancer and mismatch repair-deficient colorectal cancer

Tang, C. Z., Hartley, G. P., Couillault, C., Yuan, Y., Lin, H. K., Nicholas, C., Srinivasamani, A., Dai, J. Y., Dumbrava, E. E. I., Fu, S., et al. (2024) · BMJ Oncology · doi:10.1136/bmjonc-2023-000133 · Cited by 14

Inflammation Mediated Hepcidin-Ferroportin Pathway and Its Therapeutic Window in Breast Cancer

Shibabaw, T., Teferi, B., Molla, M. D., & Ayelign, B. (2020) · Breast Cancer Targets and Therapy · doi:10.2147/bctt.s276404 · Cited by 15

What Do We Have to Know about PD-L1 Expression in Prostate Cancer? A Systematic Literature Review. Part 4: Experimental Treatments in Pre-Clinical Studies (Cell Lines and Mouse Models)

Palicelli, A., Croci, S., Bisagni, A., Zanetti, E., de Biase, D., Melli, B., Sanguedolce, F., Ragazzi, M., Zanelli, M., Chaux, A., et al. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms222212297 · Cited by 14

A critical update on the strategies towards small molecule inhibitors targeting Serine/arginine-rich (SR) proteins and Serine/arginine-rich proteins related kinases in alternative splicing

Tang, J., Xie, Y., Huang, J., Zhang, L., Jiang, W., Li, Z., & Bian, J. (2022) · Bioorganic & Medicinal Chemistry · doi:10.1016/j.bmc.2022.116921 · Cited by 15

Synthesis and evaluation of naphthalene derivatives as potent STAT3 inhibitors and agents against triple-negative breast cancer growth and metastasis

Yang, Z., Xu, H., Yang, Y., Duan, C., Zhang, P., Wang, Y., Fu, K., Shen, Y., & Xu, M. X. (2022) · Breast Cancer Research and Treatment · doi:10.1007/s10549-022-06790-2 · Cited by 14

Targeting Smyd3 by next-generation antisense oligonucleotides suppresses liver tumor growth

Kontaki, H., Koukaki, M., Vasilarou, M., Giakountis, A., Deligianni, E., Luo, X., Kim, Y., & Talianidis, I. (2021) · iScience · doi:10.1016/j.isci.2021.102473 · Cited by 14

Targeting STAT3 Signaling in COL1+ Fibroblasts Controls Colitis-Associated Cancer in Mice

Heichler, C., Schmied, A., Enderle, K., Koop, K., Murawska, M., Schmid, B., Waldner, M. J., Neurath, M. F., & Neufert, C. F. (2022) · Cancers · doi:10.3390/cancers14061472 · Cited by 13

Targeting DNA2 overcomes metabolic reprogramming in multiple myeloma

Thongon, N., Ma, F., Baran, N., Lockyer, P. P., Liu, J., Jackson, C. E., Rose, A. M., Furudate, K., Wildeman, B., Marchesini, M., et al. (2024) · Nature Communications · doi:10.1038/s41467-024-45350-8 · Cited by 14

E-cadherin downregulation sensitizes PTEN-mutant tumors to PI3Kβ silencing

Millán‐Uclés, Á., Zuluaga, S., Marqués, M., Vallejo-Díaz, J., Sanz, L., Cariaga-Martínez, A. E., Real, F. X., & Carrera, A. C. (2016) · Oncotarget · doi:10.18632/oncotarget.13414 · Cited by 13

New twists on long noncoding RNAs: from mobile elements to motile cancer cells

Nguyen, T. M., Alchalabi, S., Adewunmi, O., Ropri, A. S., Herschkowitz, J. I., & Rosen, J. M. (2020) · RNA Biology · doi:10.1080/15476286.2020.1760535 · Cited by 13

Prostate cancer racial, socioeconomic, geographic disparities: targeting the genomic landscape and splicing events in search for diagnostic, prognostic and therapeutic targets.

Marima, R. M., Hull, R., Mathabe, K., Setlai, B., Batra, J., Sartor, O. A., Mehrotra, R., & Dlamini, Z. (2021) · PubMed Central · Cited by 13

Novel Therapeutic Approaches Targeting Post-Translational Modifications in Lung Cancer

Baietti, M. F., & Sewduth, R. N. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15010206 · Cited by 13

Abstract LB-227: Preclinical pharmacology and clinical efficacy of AZD9150 (ISIS-STAT3Rx), a potent next-generation antisense oligonucleotide inhibitor of STAT3

Hong, D. S., Kim, Y., Younes, A., Nemunaitis, J. J., Fowler, N. H., Hsu, J., Zhou, T., Fayad, L., Zhang, N., Piha-Paul, S. A., et al. (2014) · Cancer Research · doi:10.1158/1538-7445.am2014-lb-227 · Cited by 5

Abstract LB-317: Potent in vivo pharmacology of AZD9150, a next-generation, constrained ethyl-modified antisense oligonucleotide targeting STAT3 in multiple preclinical cancer models.

Kim, Y., Hsu, J., Zhou, T., Zhang, N., Woessner, R. D., Nadella, M. V. P., Lawson, D., Reimer, C., He, G., Schmidt, J., et al. (2013) · Cancer Research · doi:10.1158/1538-7445.am2013-lb-317 · Cited by 2

Blocking transcription in tumors, STAT

Nusinovich, Y. A. (2015) · Science Signaling · doi:10.1126/scisignal.aad9035

Strong pharmacological activity of locally administered next-generation antisense oligonucleotides (ASOs) in orthotopic lung cancer mouse models

Jo, M., Schmidt, J., Zhou, T., Wu, X., Crosby, J. R., Brand, E., Kim, Y., & MacLeod, A. R. (2016) · Journal of Thoracic Oncology · doi:10.1016/j.jtho.2015.12.043 · Cited by 1

Abstract C136: Exploring the functional contribution of STAT3 activity in tumor and tumor-associated stromal cells with species-selective generation 2.5 antisense oligonucleotides in preclinical cancer models.

Hsu, J., He, G., Bhattacharjee, G., Zhou, T., Kim, Y., Monia, B. P., & MacLeod, R. (2011) · Molecular Cancer Therapeutics · doi:10.1158/1535-7163.targ-11-c136