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To date, most approved ASO-based RNA therapies target non-malignant disorders such as neurodegenerative diseases, and only a single therapy in this class has been approved for cancer. Notably, nearly half of existing RNA therapeutics act by modulating RNA splicing. Given the growing evidence implicating aberrant RNA splicing in cancer pathogenesis, the development of ASO-based therapeutics for oncologic indications is expected to accelerate. More than 250 clinical trials have evaluated oligonucleotide agents targeting diverse cancer-associated molecules, with several showing encouraging early results. In this review, we summarize recent advances in understanding cancer biology relevant to ASO-based therapies and highlight ongoing progress in the development of RNA-targeted approaches for cancer treatment.<\/jats:p>","DOI":"10.1093\/jjco\/hyag017","type":"journal-article","created":{"date-parts":[[2026,1,19]],"date-time":"2026-01-19T12:18:50Z","timestamp":1768825130000},"page":"503-517","source":"Crossref","is-referenced-by-count":4,"title":["Advances in antisense oligonucleotide treatment for cancer"],"prefix":"10.1093","volume":"56","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7664-613X","authenticated-orcid":false,"given":"Lu","family":"Zhu","sequence":"first","affiliation":[{"name":"Division of Cancer RNA Research, National Cancer Center Research Institute , 5-1-1 Tsukiji, Chuo-ku, Tokyo 104-0045 ,","place":["Japan"]},{"name":"Department of Hematology and Oncology, Graduate School of Medicine, The University of Tokyo , 7-3-1, Hongo, Bunkyo-ku, Tokyo 113-0033 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