KR20230010231A - 생체내 형질도입을 위한 벡터 및 방법 - Google Patents

생체내 형질도입을 위한 벡터 및 방법 Download PDF

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KR20230010231A
KR20230010231A KR1020227043191A KR20227043191A KR20230010231A KR 20230010231 A KR20230010231 A KR 20230010231A KR 1020227043191 A KR1020227043191 A KR 1020227043191A KR 20227043191 A KR20227043191 A KR 20227043191A KR 20230010231 A KR20230010231 A KR 20230010231A
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mir
cells
cell
domain
receptor
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더글라스 제이. 졸리
데렉 쥐. 오스터태그
노리유키 가사하라
카를로스 이바네츠
코넬리아 에이. 벤틀리
모흐드 무스타크 후사인
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아빈투스 바이오, 인코포레이티드
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025033948A1 (ko) * 2023-08-07 2025-02-13 마루테라퓨틱스 주식회사 유전자 고효율 발현이 가능한 비바이러스성 듀얼 플라스미드 벡터와 이를 발현하는 줄기세포 및 분화된 조혈계 세포

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN116178567B (zh) * 2022-10-28 2025-01-28 四川大学华西医院 一种靶向TGFβRII的嵌合抗原受体及其用途
WO2024254346A1 (en) * 2023-06-07 2024-12-12 The Broad Institute, Inc. Engineered viral like particles (evlps) for the selective transduction of target cells
CN116814664B (zh) * 2023-08-25 2023-12-12 中国医学科学院肿瘤医院 一种扩展肿瘤识别表位的cea嵌合抗原受体t细胞的制备与应用
CN116814595B (zh) * 2023-08-30 2023-11-28 江苏申基生物科技有限公司 一种腺苷脱氨酶突变体及其固定化
WO2026149447A1 (zh) * 2025-01-07 2026-07-16 深圳市济因生物科技有限公司 一种颗粒及其应用

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5693508A (en) * 1994-11-08 1997-12-02 Chang; Lung-Ji Retroviral expression vectors containing MoMLV/CMV-IE/HIV-TAR chimeric long terminal repeats
US5681746A (en) * 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
US7001733B1 (en) * 1998-05-12 2006-02-21 Rigel Pharmaceuticals, Inc. Methods and compositions for screening for modulations of IgE synthesis, secretion and switch rearrangement
WO2001091802A1 (en) * 2000-05-30 2001-12-06 Baylor College Of Medicine Chimeric viral vectors for gene therapy
CN1860232A (zh) * 2003-09-30 2006-11-08 莱克康遗传公司 鉴别基因功能的方法和组合物
WO2009114725A2 (en) * 2008-03-12 2009-09-17 Children's Hospital Medical Center Mobilization of hematopoietic stem cells
US20110098346A1 (en) * 2008-05-19 2011-04-28 Agency For Science, Technology And Research Nucleic acid molecule and method of targeting gene expression to gliomas
EP2294218A2 (en) * 2008-06-02 2011-03-16 New York University Compositions and methods for diagnosis, prognosis and treatment of mesothelioma
US20110020814A1 (en) * 2009-06-05 2011-01-27 Ipierian, Inc. Methods and compositions for selection of stem cells
EP3546583A1 (en) * 2009-06-17 2019-10-02 Tocagen Inc. Producer cells for replication competent retroviral vectors
US9839696B2 (en) * 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US9642921B2 (en) * 2012-12-20 2017-05-09 Tocagen Inc. Cancer combination therapy and recombinant vectors
CA2912389C (en) * 2013-06-10 2020-01-28 The General Hospital Corporation D/B/A Massachusetts General Hospital Methods and compositions for reducing immunosupression by tumor cells
JP2016526920A (ja) * 2013-08-05 2016-09-08 トカジェン インコーポレーテッド 最適化ａ−バルジを有する組換えベクター
US10383929B2 (en) * 2014-12-12 2019-08-20 Bluebird Bio, Inc. BCMA chimeric antigen receptors
AU2016317936A1 (en) * 2015-09-04 2018-03-08 Tocagen Inc. Recombinant vectors comprising 2A peptide
CN108884460B (zh) * 2016-03-19 2023-04-28 埃克苏马生物技术公司 淋巴细胞转导及其扩增调节的方法与组合物
US11111505B2 (en) * 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
CA3030003A1 (en) * 2016-07-08 2018-01-11 F1 Oncology, Inc. Methods and compositions for transducing lymphocytes and regulating the activity thereof
DK3612624T3 (da) * 2017-04-21 2024-12-09 Ospedale San Raffaele Srl Genterapi
US20180327857A1 (en) * 2017-05-09 2018-11-15 Shanghai Realgen Biotech Co., Ltd. Diagnostic biomarker and diagnostic method
WO2019025800A1 (en) * 2017-08-02 2019-02-07 Autolus Limited CELLS EXPRESSING A CHIMERIC ANTIGENIC RECEPTOR OR A MANIPULATED TCR AND COMPRISING A SELECTIVELY EXPRESSED SELECTIVE NUCLEOTIDE SEQUENCE
KR20210063348A (ko) * 2018-08-28 2021-06-01 이뮤노테크 바이오팜 씨오., 엘티디. 개선된 치료용 t 세포

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025033948A1 (ko) * 2023-08-07 2025-02-13 마루테라퓨틱스 주식회사 유전자 고효율 발현이 가능한 비바이러스성 듀얼 플라스미드 벡터와 이를 발현하는 줄기세포 및 분화된 조혈계 세포

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