دورية أكاديمية
أعرض في EDS

AAV-mediated delivery of a Sleeping Beauty transposon and an mRNA-encoded transposase for the engineering of therapeutic immune cells.

التفاصيل البيبلوغرافية
العنوان: AAV-mediated delivery of a Sleeping Beauty transposon and an mRNA-encoded transposase for the engineering of therapeutic immune cells.
المؤلفون: Ye L; Department of Genetics, Yale University School of Medicine, New Haven, CT, USA.; System Biology Institute, Yale University, West Haven, CT, USA.; Center for Cancer Systems Biology, Yale University, West Haven, CT, USA.; Institute of Modern Biology, Nanjing University, Nanjing, China., Lam SZ; Department of Genetics, Yale University School of Medicine, New Haven, CT, USA.; System Biology Institute, Yale University, West Haven, CT, USA.; Center for Cancer Systems Biology, Yale University, West Haven, CT, USA., Yang L; Department of Genetics, Yale University School of Medicine, New Haven, CT, USA.; System Biology Institute, Yale University, West Haven, CT, USA.; Center for Cancer Systems Biology, Yale University, West Haven, CT, USA.; Molecular Cell Biology, Genetics, and Development Program, Yale University, New Haven, CT, USA., Suzuki K; Department of Genetics, Yale University School of Medicine, New Haven, CT, USA.; System Biology Institute, Yale University, West Haven, CT, USA.; Center for Cancer Systems Biology, Yale University, West Haven, CT, USA., Zou Y; Department of Genetics, Yale University School of Medicine, New Haven, CT, USA.; System Biology Institute, Yale University, West Haven, CT, USA.; Center for Cancer Systems Biology, Yale University, West Haven, CT, USA., Lin Q; Department of Genetics, Yale University School of Medicine, New Haven, CT, USA.; System Biology Institute, Yale University, West Haven, CT, USA.; Center for Cancer Systems Biology, Yale University, West Haven, CT, USA., Zhang Y; Department of Genetics, Yale University School of Medicine, New Haven, CT, USA.; System Biology Institute, Yale University, West Haven, CT, USA.; Center for Cancer Systems Biology, Yale University, West Haven, CT, USA., Clark P; Department of Genetics, Yale University School of Medicine, New Haven, CT, USA.; System Biology Institute, Yale University, West Haven, CT, USA.; Center for Cancer Systems Biology, Yale University, West Haven, CT, USA., Peng L; Department of Genetics, Yale University School of Medicine, New Haven, CT, USA.; System Biology Institute, Yale University, West Haven, CT, USA.; Center for Cancer Systems Biology, Yale University, West Haven, CT, USA., Chen S; Department of Genetics, Yale University School of Medicine, New Haven, CT, USA. sidi.chen@yale.edu.; System Biology Institute, Yale University, West Haven, CT, USA. sidi.chen@yale.edu.; Center for Cancer Systems Biology, Yale University, West Haven, CT, USA. sidi.chen@yale.edu.; Molecular Cell Biology, Genetics, and Development Program, Yale University, New Haven, CT, USA. sidi.chen@yale.edu.; Immunobiology Program, Yale University, New Haven, CT, USA. sidi.chen@yale.edu.; Yale Comprehensive Cancer Center, Yale University School of Medicine, New Haven, CT, USA. sidi.chen@yale.edu.; Yale Stem Cell Center, Yale University School of Medicine, New Haven, CT, USA. sidi.chen@yale.edu.; Yale Center for Biomedical Data Science, Yale University School of Medicine, New Haven, CT, USA. sidi.chen@yale.edu.
المصدر: Nature biomedical engineering [Nat Biomed Eng] 2024 Feb; Vol. 8 (2), pp. 132-148. Date of Electronic Publication: 2023 Jul 10.
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: Springer Nature Country of Publication: England NLM ID: 101696896 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2157-846X (Electronic) Linking ISSN: 2157846X NLM ISO Abbreviation: Nat Biomed Eng Subsets: MEDLINE
أسماء مطبوعة: Publication: London : Springer Nature
Original Publication: [London] : Macmillan Publishers Limited, [2016]-
مواضيع طبية MeSH: Transposases*/genetics , Transposases*/metabolism , Dependovirus*/genetics, DNA Transposable Elements/genetics ; RNA, Messenger/genetics ; Gene Transfer Techniques
مستخلص: Engineering cells for adoptive therapy requires overcoming limitations in cell viability and, in the efficiency of transgene delivery, the duration of transgene expression and the stability of genomic integration. Here we report a gene-delivery system consisting of a Sleeping Beauty (SB) transposase encoded into a messenger RNA delivered by an adeno-associated virus (AAV) encoding an SB transposon that includes the desired transgene, for mediating the permanent integration of the transgene. Compared with lentiviral vectors and with the electroporation of plasmids of transposon DNA or minicircle DNA, the gene-delivery system, which we named MAJESTIC (for 'mRNA AAV-SB joint engineering of stable therapeutic immune cells'), offers prolonged transgene expression, as well as higher transgene expression, therapeutic-cell yield and cell viability. MAJESTIC can deliver chimeric antigen receptors (CARs) into T cells (which we show lead to strong anti-tumour activity in vivo) and also transduce natural killer cells, myeloid cells and induced pluripotent stem cells with bi-specific CARs, kill-switch CARs and synthetic T-cell receptors.
(© 2023. The Author(s), under exclusive licence to Springer Nature Limited.)
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معلومات مُعتمدة: DP2CA238295 U.S. Department of Health & Human Services | National Institutes of Health (NIH); RF1 DA048811 United States DA NIDA NIH HHS; R01 CA231112 United States CA NCI NIH HHS; DP2 CA238295 United States CA NCI NIH HHS; R33CA225498 U.S. Department of Health & Human Services | National Institutes of Health (NIH); R33 CA225498 United States CA NCI NIH HHS
المشرفين على المادة: EC 2.7.7.- (Transposases)
0 (DNA Transposable Elements)
0 (RNA, Messenger)
تواريخ الأحداث: Date Created: 20230710 Date Completed: 20240222 Latest Revision: 20240815
رمز التحديث: 20240815
مُعرف محوري في PubMed: PMC11320892
DOI: 10.1038/s41551-023-01058-6
PMID: 37430157
قاعدة البيانات: MEDLINE
الوصف
تدمد:2157-846X
DOI:10.1038/s41551-023-01058-6