Tumor-specific AAV delivery of interleukin-12 enhances antitumor immunity and safety in ovarian cancer xenograft mouse model

Interleukin-12 (IL-12) is a promising pro-inflammatory cytokine for cancer immunotherapy, but its toxicity and short half-life in serum limit its clinical application. Tumor-targeted delivery of IL-12 by fusion with either antibody or secretion by chimeric antigen receptor T (CAR-T) cells showed red...

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Main Authors: Chuyuan Chen, Yongji Jiang, Chuan Feng, Qingyun Zhou, Xingrong Luo, Lili Cai, Lei Zhao
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Oncology
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Online Access:http://www.sciencedirect.com/science/article/pii/S2950329925000712
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author Chuyuan Chen
Yongji Jiang
Chuan Feng
Qingyun Zhou
Xingrong Luo
Lili Cai
Lei Zhao
author_facet Chuyuan Chen
Yongji Jiang
Chuan Feng
Qingyun Zhou
Xingrong Luo
Lili Cai
Lei Zhao
author_sort Chuyuan Chen
collection DOAJ
description Interleukin-12 (IL-12) is a promising pro-inflammatory cytokine for cancer immunotherapy, but its toxicity and short half-life in serum limit its clinical application. Tumor-targeted delivery of IL-12 by fusion with either antibody or secretion by chimeric antigen receptor T (CAR-T) cells showed reduced systematic toxicity; however, the poor tumor microenvironment (TME) response or the lack of systematic IL-12 regulation still remains risk of low efficacy or high toxicity. Here, we developed TME-specific delivery of IL-12 by a tumor-targeted adeno-associated virus 9 (tAAV9). The tAAV9 was formed by an anti-folate receptor 1 (anti-FOLR1) antibody fragment conjugated with AAV9 via highly efficient Spy-ligation. With targeted infection of FOLR1+ cells in vivo, intravenous (i.v.) administration of tAAV9 specifically delivered IL-12 (tAAV9-IL-12) to TME and significantly suppressed tumor progression with favorable safety profile compared with rAAV9 (recombinant wild-type AAV9) delivery. Moreover, the IL-12 level in the serum was decreased significantly with the suppression of tAAV9-IL-12-infected tumor cell, so that generates promising negative feedback to ensure the safety profile.
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publishDate 2025-06-01
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series Molecular Therapy: Oncology
spelling doaj-art-258969ffc4b9428caa20782a52e3554c2025-08-20T02:03:25ZengElsevierMolecular Therapy: Oncology2950-32992025-06-0133220100210.1016/j.omton.2025.201002Tumor-specific AAV delivery of interleukin-12 enhances antitumor immunity and safety in ovarian cancer xenograft mouse modelChuyuan Chen0Yongji Jiang1Chuan Feng2Qingyun Zhou3Xingrong Luo4Lili Cai5Lei Zhao6Department of Gene Therapy, Cure Genetics Co., LTD, Suzhou, ChinaDepartment of Gene Therapy, Cure Genetics Co., LTD, Suzhou, ChinaDivision of Research & Development, Department of Cell Therapy, Cure Genetics Co., LTD, Suzhou, ChinaDepartment of Gene Therapy, Cure Genetics Co., LTD, Suzhou, ChinaDepartment of Gene Therapy, Cure Genetics Co., LTD, Suzhou, ChinaDepartment of Gene Therapy, Cure Genetics Co., LTD, Suzhou, ChinaDepartment of Gene Therapy, Cure Genetics Co., LTD, Suzhou, China; Corresponding author: Lei Zhao, Department of Gene Therapy, Cure Genetics Co., LTD, Suzhou, China.Interleukin-12 (IL-12) is a promising pro-inflammatory cytokine for cancer immunotherapy, but its toxicity and short half-life in serum limit its clinical application. Tumor-targeted delivery of IL-12 by fusion with either antibody or secretion by chimeric antigen receptor T (CAR-T) cells showed reduced systematic toxicity; however, the poor tumor microenvironment (TME) response or the lack of systematic IL-12 regulation still remains risk of low efficacy or high toxicity. Here, we developed TME-specific delivery of IL-12 by a tumor-targeted adeno-associated virus 9 (tAAV9). The tAAV9 was formed by an anti-folate receptor 1 (anti-FOLR1) antibody fragment conjugated with AAV9 via highly efficient Spy-ligation. With targeted infection of FOLR1+ cells in vivo, intravenous (i.v.) administration of tAAV9 specifically delivered IL-12 (tAAV9-IL-12) to TME and significantly suppressed tumor progression with favorable safety profile compared with rAAV9 (recombinant wild-type AAV9) delivery. Moreover, the IL-12 level in the serum was decreased significantly with the suppression of tAAV9-IL-12-infected tumor cell, so that generates promising negative feedback to ensure the safety profile.http://www.sciencedirect.com/science/article/pii/S2950329925000712MT: Regular Issuecell specific AAVinterleukin-12tumor microenvironmentsolid tumorgene therapy
spellingShingle Chuyuan Chen
Yongji Jiang
Chuan Feng
Qingyun Zhou
Xingrong Luo
Lili Cai
Lei Zhao
Tumor-specific AAV delivery of interleukin-12 enhances antitumor immunity and safety in ovarian cancer xenograft mouse model
Molecular Therapy: Oncology
MT: Regular Issue
cell specific AAV
interleukin-12
tumor microenvironment
solid tumor
gene therapy
title Tumor-specific AAV delivery of interleukin-12 enhances antitumor immunity and safety in ovarian cancer xenograft mouse model
title_full Tumor-specific AAV delivery of interleukin-12 enhances antitumor immunity and safety in ovarian cancer xenograft mouse model
title_fullStr Tumor-specific AAV delivery of interleukin-12 enhances antitumor immunity and safety in ovarian cancer xenograft mouse model
title_full_unstemmed Tumor-specific AAV delivery of interleukin-12 enhances antitumor immunity and safety in ovarian cancer xenograft mouse model
title_short Tumor-specific AAV delivery of interleukin-12 enhances antitumor immunity and safety in ovarian cancer xenograft mouse model
title_sort tumor specific aav delivery of interleukin 12 enhances antitumor immunity and safety in ovarian cancer xenograft mouse model
topic MT: Regular Issue
cell specific AAV
interleukin-12
tumor microenvironment
solid tumor
gene therapy
url http://www.sciencedirect.com/science/article/pii/S2950329925000712
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