JP4861314B2 - 非組み込み型且つ非複製型組換えレンチウイルス、その調製および使用 - Google Patents

非組み込み型且つ非複製型組換えレンチウイルス、その調製および使用 Download PDF

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JP4861314B2
JP4861314B2 JP2007517365A JP2007517365A JP4861314B2 JP 4861314 B2 JP4861314 B2 JP 4861314B2 JP 2007517365 A JP2007517365 A JP 2007517365A JP 2007517365 A JP2007517365 A JP 2007517365A JP 4861314 B2 JP4861314 B2 JP 4861314B2
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sequence
lentiviral
integrase
region
cells
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JP2008503230A5 (enExample
JP2008503230A (ja
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マル，ジャック
セルゲーラ，シェ
フィリップ，ステファニー
サルキス，シャムジ
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Centre National de la Recherche Scientifique CNRS
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JP2007517365A 2004-06-25 2005-06-24 非組み込み型且つ非複製型組換えレンチウイルス、その調製および使用 Expired - Fee Related JP4861314B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
FR0407017A FR2872170B1 (fr) 2004-06-25 2004-06-25 Lentivirus non interactif et non replicatif, preparation et utilisations
FR0407017 2004-06-25
PCT/FR2005/001604 WO2006010834A1 (fr) 2004-06-25 2005-06-24 Lentivirus non integratif et non replicatif, preparation et utilisations

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JP2008503230A JP2008503230A (ja) 2008-02-07
JP2008503230A5 JP2008503230A5 (enExample) 2008-08-07
JP4861314B2 true JP4861314B2 (ja) 2012-01-25

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JP2007517365A Expired - Fee Related JP4861314B2 (ja) 2004-06-25 2005-06-24 非組み込み型且つ非複製型組換えレンチウイルス、その調製および使用

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US (1) US8119119B2 (enExample)
EP (1) EP1761635B1 (enExample)
JP (1) JP4861314B2 (enExample)
CN (1) CN101023177A (enExample)
AT (1) ATE524554T1 (enExample)
AU (1) AU2005266221B2 (enExample)
CA (1) CA2579753C (enExample)
FR (1) FR2872170B1 (enExample)
IL (1) IL179740A0 (enExample)
WO (1) WO2006010834A1 (enExample)

Families Citing this family (112)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0526211D0 (en) * 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
WO2007091066A1 (en) * 2006-02-07 2007-08-16 Ucl Business Plc Applications of non-integrating lentiviral vectors
AU2013203404B2 (en) * 2007-08-03 2016-10-13 Centre National De La Recherche Scientifique (Cnrs) Lentiviral gene transfer vectors and their medicinal applications
EP2020444B1 (en) * 2007-08-03 2017-05-31 Institut Pasteur Defective non-integrative lentiviral transfer vectors for vaccines
CN102083462B (zh) 2007-08-03 2015-02-18 巴斯德研究院 慢病毒基因转移载体及其医学应用
CA2703045C (en) 2007-10-25 2017-02-14 Sangamo Biosciences, Inc. Methods and compositions for targeted integration
US20110142880A1 (en) * 2008-03-28 2011-06-16 Franck Yann Lemiale Lentivirus-based immunogenic vectors
WO2009131706A1 (en) * 2008-04-26 2009-10-29 Yung-Nien Chang Site-specific-integration lentiviral vectors
WO2010105251A2 (en) * 2009-03-13 2010-09-16 Lentigen Corporation Non-integrating retroviral vector vaccines
CA2756833C (en) 2009-04-09 2019-11-19 Sangamo Biosciences, Inc. Targeted integration into stem cells
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
CA2788850C (en) 2010-02-09 2019-06-25 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
DK2385107T3 (en) 2010-05-03 2016-12-12 Pasteur Institut Lentiviral vector-based immunological compounds against malaria
WO2012152912A1 (en) 2011-05-12 2012-11-15 Newvectys Genetically modified pig as a cancer prone model
ES2651922T3 (es) 2011-11-08 2018-01-30 Institut Pasteur Polipéptidos de afinidad elevada para MAST2 y usos de los mismos
GB201202516D0 (en) 2012-02-13 2012-03-28 Ucl Business Plc Materials and methods relating to packaging cell lines
CN104583231B (zh) 2012-03-30 2021-02-26 免疫设计公司 具有对表达dc-sign的细胞改善的转导效率的慢病毒载体粒子
US9713635B2 (en) 2012-03-30 2017-07-25 Immune Design Corp. Materials and methods for producing improved lentiviral vector particles
JP2016501531A (ja) 2012-12-12 2016-01-21 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作および治療適用のための系、方法および組成物の送達、エンジニアリングおよび最適化
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
ES2701749T3 (es) 2012-12-12 2019-02-25 Broad Inst Inc Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos
AU2013204922B2 (en) 2012-12-20 2015-05-14 Celgene Corporation Chimeric antigen receptors
CN105518018B (zh) 2013-03-15 2020-04-03 细胞基因公司 修饰的t淋巴细胞
EP3825406A1 (en) 2013-06-17 2021-05-26 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
SG10201710487VA (en) * 2013-06-17 2018-01-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3620524A1 (en) * 2013-06-17 2020-03-11 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
EP3011030B1 (en) 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
EP3046930A1 (en) * 2013-09-17 2016-07-27 INSERM - Institut National de la Santé et de la Recherche Médicale Lentiviral vectors having a mutated integrase protein and uses thereof
EP2878667A1 (en) 2013-11-29 2015-06-03 Institut Pasteur TAL effector means useful for partial or full deletion of DNA tandem repeats
KR20160089530A (ko) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Ｈｂｖ 및 바이러스 질병 및 질환을 위한 ｃｒｉｓｐｒ­ｃａｓ 시스템 및 조성물의 전달，용도 및 치료적 적용
WO2015089462A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
BR112016013547A2 (pt) 2013-12-12 2017-10-03 Broad Inst Inc Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos
EP4219699A1 (en) 2013-12-12 2023-08-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
PL3689899T3 (pl) 2014-04-25 2022-01-31 2Seventy Bio, Inc. Chimeryczne receptory antygenowe promotora mnd
SMT202000355T1 (it) 2014-04-25 2020-09-10 Bluebird Bio Inc Metodi migliorati per produrre terapie con cellule adottive
EP3151672B1 (en) 2014-06-06 2020-11-04 Bluebird Bio, Inc. Improved t cell compositions
CA2956002C (en) 2014-07-24 2023-06-27 Bluebird Bio, Inc. Bcma chimeric antigen receptors
EP3031923A1 (en) 2014-12-11 2016-06-15 Institut Pasteur Lentiviral vector-based japanese encephalitis immunogenic composition
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
JP6671370B2 (ja) * 2014-12-12 2020-03-25 ブルーバード バイオ， インコーポレイテッド Ｂｃｍａキメラ抗原受容体
AU2015369725A1 (en) 2014-12-24 2017-06-29 Massachusetts Institute Of Technology CRISPR having or associated with destabilization domains
RU2752834C2 (ru) 2015-06-18 2021-08-09 Те Брод Инститьют, Инк. Мутации фермента crispr, уменьшающие нецелевые эффекты
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US10701165B2 (en) 2015-09-23 2020-06-30 Sensoriant, Inc. Method and system for using device states and user preferences to create user-friendly environments
WO2017060662A1 (en) * 2015-10-07 2017-04-13 The Secretary Of State For Health Method for providing a control for use in a screen for pathogenic virus
US11479755B2 (en) 2015-12-07 2022-10-25 2Seventy Bio, Inc. T cell compositions
EP3443002A4 (en) 2016-04-14 2019-12-04 Bluebird Bio, Inc. SYSTEMS FOR RECOVERED CHIMERIC ANTIGEN RECEPTORS
WO2017214938A1 (zh) * 2016-06-16 2017-12-21 毛侃琅 特异促进 bace1 基因高表达的慢病毒表达载体及应用
CA3034094A1 (en) 2016-08-16 2018-02-22 Bluebird Bio, Inc. Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use
CA3034782A1 (en) 2016-08-23 2018-03-01 Bluebird Bio, Inc. Tim3 homing endonuclease variants, compositions, and methods of use
CA3035455A1 (en) 2016-09-08 2018-03-15 Bluebird Bio, Inc. Pd-1 homing endonuclease variants, compositions, and methods of use
US11530395B2 (en) 2016-10-17 2022-12-20 2Seventy Bio, Inc. TGFBetaR2 endonuclease variants, compositions, and methods of use
JP7093346B2 (ja) 2016-11-04 2022-06-29 ２セブンティ バイオ インコーポレイテッド 抗ｂｃｍａ ｃａｒ ｔ細胞組成物
JP7263235B2 (ja) 2016-11-17 2023-04-24 ２セブンティ バイオ インコーポレイテッド ＴＧＦβシグナルコンバーター
US20200071721A1 (en) * 2016-12-06 2020-03-05 Bluebird Bio, Inc. Gene therapy for mucopolysaccharidosis, type ii
MX2019006552A (es) * 2016-12-06 2019-10-15 Bluebird Bio Inc Terapia génica para mucopolisacaridosis de tipo i.
EP3357504A1 (en) 2017-02-02 2018-08-08 Institut Pasteur Functional screening of antigenic polypeptides - use for the identification of antigens eliciting a protective immune response and for the selection of antigens with optimal protective activity
EP3357506A1 (en) 2017-02-02 2018-08-08 Institut Pasteur Multiple malaria pre-erythrocytic antigens and their use in the elicitation of a protective immune response in a host
DK3583203T5 (da) 2017-02-15 2024-09-02 2Seventy Bio Inc Donorreparationstemplates multiplex-genomeditering
IL270776B2 (en) 2017-05-25 2024-11-01 Bluebird Bio Inc Cblb endonuclease variants, compositions, and methods of use
US11779654B2 (en) 2017-10-04 2023-10-10 2Seventy Bio, Inc. PCSK9 endonuclease variants, compositions, and methods of use
FI3633040T3 (fi) 2017-12-22 2023-08-03 Oxford Biomedica Ltd Retrovirusvektori
DE102018100967B4 (de) 2018-01-17 2019-08-14 Immatics US, Inc. Verfahren zur feststellung der wirksamkeit von viralen vektoren
DE102018010282A1 (de) 2018-01-17 2019-07-18 Immatics US, Inc. Verfahren zur Feststellung der Wirksamkeit von viralen Vektoren
BR112020025439A2 (pt) 2018-06-14 2021-03-16 Bluebird Bio, Inc. Receptores de antígenos quiméricos cd79a
EP3820515A1 (en) 2018-07-11 2021-05-19 Celgene Corporation Uses of anti-bcma chimeric antigen receptors
CN113164556A (zh) 2018-08-30 2021-07-23 特纳亚治疗股份有限公司 用心肌蛋白和ascl1进行的心脏细胞重编程
WO2020072059A1 (en) 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
EP3893922A4 (en) 2018-12-10 2022-09-14 2seventy bio, Inc. VARIANTS OF ENDONUCLEASE HOMING
SG11202110607WA (en) 2019-04-01 2021-10-28 Tenaya Therapeutics Inc Adeno-associated virus with engineered capsid
WO2021007515A1 (en) 2019-07-11 2021-01-14 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
EP3770264A1 (en) 2019-07-22 2021-01-27 Genethon Precise integration using nuclease targeted idlv
US20220331363A1 (en) 2019-09-20 2022-10-20 Shanghai GenBase Biotechnology Co., Ltd. Bcma-targeted antibody and chimeric antigen receptor
CN115697387A (zh) 2019-11-05 2023-02-03 细胞基因公司 抗bcma嵌合抗原受体的用途
WO2021094752A1 (en) 2019-11-12 2021-05-20 Oxford Biomedica (Uk) Limited Production system
WO2021160993A1 (en) 2020-02-13 2021-08-19 Oxford Biomedica (Uk) Limited Production of lentiviral vectors
KR20220154734A (ko) 2020-03-13 2022-11-22 옥스포드 바이오메디카(유케이) 리미티드 렌티바이러스 벡터
WO2021212279A1 (zh) * 2020-04-20 2021-10-28 广东东阳光药业有限公司 慢病毒的滴度提高型转移质粒
CN111534533B (zh) * 2020-04-27 2022-04-19 北京化工大学 一种t7rna聚合酶和t7启动子的表达系统及使用其在真核生物中表达蛋白质的方法
GB202007199D0 (en) 2020-05-15 2020-07-01 Oxford Biomedica Ltd Viral vector production
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
EP3984548A1 (en) 2020-10-16 2022-04-20 Institut Pasteur Generation of lentiviral vectors enabling routing antigens to mhc-ii pathway and inducing cd4+ and cd8+ t-cell responses immune response in a host
KR20230113755A (ko) 2020-11-04 2023-08-01 셀진 코포레이션 선행 항암 알킬화제 요법을 받은 환자에서의 car t 세포 요법
GB202017725D0 (en) 2020-11-10 2020-12-23 Oxford Biomedica Ltd Method
JP2023552773A (ja) 2020-12-04 2023-12-19 セルジーン コーポレーション 炎症関連可溶性因子の阻害剤と組み合わせたキメラ抗原受容体（ｃａｒ）ｔ細胞療法の使用
CA3209285A1 (en) 2021-03-12 2022-09-15 Pierre Charneau Lentiviral vectors targeting antigens to mhc-ii pathway and inducing protective cd8+ and cd4+ t-cell immunity in a host
CA3214280A1 (en) 2021-04-16 2022-10-20 Julie Ann RYTLEWSKI T cell therapy in patients who have had prior stem cell transplant
EP4370541A1 (en) 2021-07-14 2024-05-22 2seventy bio, Inc. Engineered t cell receptors fused to binding domains from antibodies
GB202114534D0 (en) 2021-10-12 2021-11-24 Oxford Biomedica Ltd Novel viral regulatory elements
GB202114532D0 (en) 2021-10-12 2021-11-24 Oxford Biomedica Ltd Lentiviral Vectors
GB202114529D0 (en) 2021-10-12 2021-11-24 Oxford Biomedica Ltd Lentiviral vectors
GB202114528D0 (en) 2021-10-12 2021-11-24 Oxford Biomedica Ltd Lentiviral vectors
GB202114530D0 (en) 2021-10-12 2021-11-24 Oxford Biomedica Ltd Retroviral vectors
CN114181972A (zh) * 2021-11-23 2022-03-15 上海本导基因技术有限公司 适用于难治性血管新生性眼疾病基因治疗的慢病毒载体
GB202117844D0 (en) 2021-12-09 2022-01-26 Oxford Biomedica Ltd Purification method
JP2025511872A (ja) 2022-04-08 2025-04-16 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 多部位受容体及びシグナル伝達複合体
CA3246384A1 (en) 2022-04-13 2023-10-19 Fundacio Hospital Univ Vall Dhebron Institut De Recerca TREATMENT OF NEUROMUSCULAR DISEASES BY GENE THERAPY EXPRESSING THE KLOTHO PROTEIN
WO2023220641A2 (en) 2022-05-11 2023-11-16 Juno Therapeutics, Inc. Methods and uses related to t cell therapy and production of same
CN119585438A (zh) 2022-05-26 2025-03-07 再生元制药公司 用于维持慢病毒载体的组合物及其用途
GB202211935D0 (en) 2022-08-16 2022-09-28 Oxford Biomedica Ltd envelope proteins
AU2023362558A1 (en) 2022-10-21 2025-05-08 Theravectys Polynucleotides and lentiviral vectors expressing non-structural antigens of a flavivirus selected from the group of denv, zikv and yfv, inducing protective cd8+ t-cell immunity in a host
WO2024102954A1 (en) 2022-11-10 2024-05-16 Massachusetts Institute Of Technology Activation induced clipping system (aics)
WO2024200573A1 (en) 2023-03-27 2024-10-03 LAVA Therapeutics N.V. Nectin-4 binding agents and methods of use
WO2025012118A2 (en) 2023-07-07 2025-01-16 LAVA Therapeutics N.V. 5t4 binding agents and methods of use
WO2025059162A1 (en) 2023-09-11 2025-03-20 Dana-Farber Cancer Institute, Inc. Car-engager containing il-2 variants to enhance the functionality of car t cells
WO2025153530A1 (en) 2024-01-16 2025-07-24 Novo Nordisk A/S Albumin-targeted endonucleases, compositions, and methods of use
WO2025235862A1 (en) 2024-05-10 2025-11-13 Inndura Therapeutics Inc. A modified immune cell receptor comprising a target-binding domain and the extracellular domain of cd16a

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000072886A1 (en) * 1999-05-26 2000-12-07 Dana-Farber Cancer Institute, Inc. Episomally replicating lentiviral vectors
JP2000517290A (ja) * 1996-02-21 2000-12-26 リスジーウィックス，ジュリアナ 保護的及び治療的な遺伝子免疫化のための方法及び組成物
WO2002024897A2 (en) * 2000-09-22 2002-03-28 Virxsys Conditionally replicating viral vectors and their use

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3140757B2 (ja) 1989-02-06 2001-03-05 デイナ・フアーバー・キヤンサー・インステイテユート パッケージング欠陥ｈｉｖプロウイルス、細胞系及びその使用
US5665577A (en) 1989-02-06 1997-09-09 Dana-Farber Cancer Institute Vectors containing HIV packaging sequences, packaging defective HIV vectors, and uses thereof
US5981276A (en) 1990-06-20 1999-11-09 Dana-Farber Cancer Institute Vectors containing HIV packaging sequences, packaging defective HIV vectors, and uses thereof
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
ATE367447T1 (de) 1998-05-13 2007-08-15 Genetix Pharmaceuticals Inc Lentivirale verpackungszellen
AU2487300A (en) * 1998-12-31 2000-07-31 Chiron Corporation Polynucleotides encoding antigenic hiv type c polypeptides, polypeptides and uses thereof
CN101363029B (zh) 1999-10-12 2016-01-13 巴斯德研究所 慢病毒三链体dna、及含有慢病毒三链体dna的载体和重组细胞
FR2825372B1 (fr) 2001-06-01 2004-06-18 Centre Nat Rech Scient Pseudotypage des vecteurs vih-1 par des enveloppes de rhabdovirus
US20070042494A1 (en) * 2005-05-31 2007-02-22 Tal Kafri Heterologous retroviral packaging system

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2000517290A (ja) * 1996-02-21 2000-12-26 リスジーウィックス，ジュリアナ 保護的及び治療的な遺伝子免疫化のための方法及び組成物
WO2000072886A1 (en) * 1999-05-26 2000-12-07 Dana-Farber Cancer Institute, Inc. Episomally replicating lentiviral vectors
WO2002024897A2 (en) * 2000-09-22 2002-03-28 Virxsys Conditionally replicating viral vectors and their use

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