Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects

Nicotinamide N-methyltransferase (NNMT) is one of the methyltransferase family genes. It consumes S-adenosyl-l-methionine (SAM), which is required for DNA methylation and histone methylation for epigenetic regulation, to produce 1-methylnicotinamide from nicotinamide, a source of NAD+, thus affectin...

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Main Authors: Tomoaki Hara, Sikun Meng, Yuuya Kasahara, Takashi Osawa, Daisuke Motooka, Hiromichi Sato, Yasuko Arao, Yoshiko Saito, Kana Inoue, Yumiko Hamano, Yuichiro Doki, Hidetoshi Eguchi, Satoshi Obika, Hideshi Ishii
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Nucleic Acids
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Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125001027
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author Tomoaki Hara
Sikun Meng
Yuuya Kasahara
Takashi Osawa
Daisuke Motooka
Hiromichi Sato
Yasuko Arao
Yoshiko Saito
Kana Inoue
Yumiko Hamano
Yuichiro Doki
Hidetoshi Eguchi
Satoshi Obika
Hideshi Ishii
author_facet Tomoaki Hara
Sikun Meng
Yuuya Kasahara
Takashi Osawa
Daisuke Motooka
Hiromichi Sato
Yasuko Arao
Yoshiko Saito
Kana Inoue
Yumiko Hamano
Yuichiro Doki
Hidetoshi Eguchi
Satoshi Obika
Hideshi Ishii
author_sort Tomoaki Hara
collection DOAJ
description Nicotinamide N-methyltransferase (NNMT) is one of the methyltransferase family genes. It consumes S-adenosyl-l-methionine (SAM), which is required for DNA methylation and histone methylation for epigenetic regulation, to produce 1-methylnicotinamide from nicotinamide, a source of NAD+, thus affecting energy metabolism and epigenetics. Recent studies have shown that NNMT is highly expressed in cancer tissues, mainly in the stroma, and worsens prognosis. Therefore, NNMT is attracting attention as a new target for cancer therapy. In this study, we generated 2′,4′-BNA/LNA-modified gapmer phosphorothioate antisense oligonucleotides that inhibit NNMT expression and examined their antitumor effects. The antisense oligonucleotide candidates were finally narrowed down to eight sequences, and when they were examined for their inhibitory effect on NNMT expression in cancer cells, all of the sequences showed inhibitory effects. The most effective one was conjugated with a small molecule compound that targets the stroma of cancer tissues. The antitumor effect was examined in a mouse model of cancer cell transplantation, and the antitumor effect was enhanced in the group treated with the antisense oligonucleotide. These results indicate that NNMT antisense oligonucleotide drugs targeting the stroma are promising as novel anticancer agents.
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series Molecular Therapy: Nucleic Acids
spelling doaj-art-de280e449737456b819fbc76cb6dd40b2025-08-20T03:09:16ZengElsevierMolecular Therapy: Nucleic Acids2162-25312025-06-0136210254810.1016/j.omtn.2025.102548Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effectsTomoaki Hara0Sikun Meng1Yuuya Kasahara2Takashi Osawa3Daisuke Motooka4Hiromichi Sato5Yasuko Arao6Yoshiko Saito7Kana Inoue8Yumiko Hamano9Yuichiro Doki10Hidetoshi Eguchi11Satoshi Obika12Hideshi Ishii13Department of Medical Data Science, Center of Medical Innovation and Translational Research, Osaka University Graduate School of Medicine, Osaka 565-0871, JapanDepartment of Medical Data Science, Center of Medical Innovation and Translational Research, Osaka University Graduate School of Medicine, Osaka 565-0871, JapanNational Institutes of Biomedical Innovation, Health and Nutrition, Osaka 567-0085, JapanGraduate School of Pharmaceutical Sciences, Osaka University, Osaka 565-0871, JapanGenome Information Research Center, Research Institute for Microbial Diseases, Osaka University, Osaka 565-0871, JapanDepartment of Medical Data Science, Center of Medical Innovation and Translational Research, Osaka University Graduate School of Medicine, Osaka 565-0871, Japan; Department of Gastroenterological Surgery, Osaka University Graduate School of Medicine, Osaka 565-0871, JapanDepartment of Medical Data Science, Center of Medical Innovation and Translational Research, Osaka University Graduate School of Medicine, Osaka 565-0871, JapanDepartment of Medical Data Science, Center of Medical Innovation and Translational Research, Osaka University Graduate School of Medicine, Osaka 565-0871, JapanDepartment of Medical Data Science, Center of Medical Innovation and Translational Research, Osaka University Graduate School of Medicine, Osaka 565-0871, JapanDepartment of Medical Data Science, Center of Medical Innovation and Translational Research, Osaka University Graduate School of Medicine, Osaka 565-0871, JapanDepartment of Gastroenterological Surgery, Osaka University Graduate School of Medicine, Osaka 565-0871, JapanDepartment of Gastroenterological Surgery, Osaka University Graduate School of Medicine, Osaka 565-0871, JapanGraduate School of Pharmaceutical Sciences, Osaka University, Osaka 565-0871, Japan; Corresponding author: Satoshi Obika, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan.Department of Medical Data Science, Center of Medical Innovation and Translational Research, Osaka University Graduate School of Medicine, Osaka 565-0871, Japan; Corresponding author: Hideshi Ishii, Department of Medical Data Science, Center of Medical Innovation and Translational Research, Osaka University Graduate School of Medicine, Osaka, Japan.Nicotinamide N-methyltransferase (NNMT) is one of the methyltransferase family genes. It consumes S-adenosyl-l-methionine (SAM), which is required for DNA methylation and histone methylation for epigenetic regulation, to produce 1-methylnicotinamide from nicotinamide, a source of NAD+, thus affecting energy metabolism and epigenetics. Recent studies have shown that NNMT is highly expressed in cancer tissues, mainly in the stroma, and worsens prognosis. Therefore, NNMT is attracting attention as a new target for cancer therapy. In this study, we generated 2′,4′-BNA/LNA-modified gapmer phosphorothioate antisense oligonucleotides that inhibit NNMT expression and examined their antitumor effects. The antisense oligonucleotide candidates were finally narrowed down to eight sequences, and when they were examined for their inhibitory effect on NNMT expression in cancer cells, all of the sequences showed inhibitory effects. The most effective one was conjugated with a small molecule compound that targets the stroma of cancer tissues. The antitumor effect was examined in a mouse model of cancer cell transplantation, and the antitumor effect was enhanced in the group treated with the antisense oligonucleotide. These results indicate that NNMT antisense oligonucleotide drugs targeting the stroma are promising as novel anticancer agents.http://www.sciencedirect.com/science/article/pii/S2162253125001027MT: Oligonucleotides: Therapies and Applicationsantisense oligonucleotideNNMTFAPBNA/LNAFAP-binding small-molecule-conjugated ASO and antitumor
spellingShingle Tomoaki Hara
Sikun Meng
Yuuya Kasahara
Takashi Osawa
Daisuke Motooka
Hiromichi Sato
Yasuko Arao
Yoshiko Saito
Kana Inoue
Yumiko Hamano
Yuichiro Doki
Hidetoshi Eguchi
Satoshi Obika
Hideshi Ishii
Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects
Molecular Therapy: Nucleic Acids
MT: Oligonucleotides: Therapies and Applications
antisense oligonucleotide
NNMT
FAP
BNA/LNA
FAP-binding small-molecule-conjugated ASO and antitumor
title Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects
title_full Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects
title_fullStr Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects
title_full_unstemmed Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects
title_short Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects
title_sort antisense oligonucleotide targeting nicotinamide n methyltransferase exhibits antitumor effects
topic MT: Oligonucleotides: Therapies and Applications
antisense oligonucleotide
NNMT
FAP
BNA/LNA
FAP-binding small-molecule-conjugated ASO and antitumor
url http://www.sciencedirect.com/science/article/pii/S2162253125001027
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