JP5761996B2 - 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ - Google Patents

合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ Download PDF

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JP5761996B2
JP5761996B2 JP2010532293A JP2010532293A JP5761996B2 JP 5761996 B2 JP5761996 B2 JP 5761996B2 JP 2010532293 A JP2010532293 A JP 2010532293A JP 2010532293 A JP2010532293 A JP 2010532293A JP 5761996 B2 JP5761996 B2 JP 5761996B2
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meganuclease
sequence
chain
laglidadg
nucleic acid
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JP2011501971A (ja
JP2011501971A5 (enExample
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スミス，ジェイムス，ジェファーソン
ジャンツ，デレック
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プレシジョン バイオサイエンシズ，インク．
プレシジョン バイオサイエンシズ，インク．
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
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    • A61P33/00—Antiparasitic agents
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12N2800/00—Nucleic acids vectors
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)

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  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
JP2010532293A 2007-10-31 2008-10-31 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ Active JP5761996B2 (ja)

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US124707P 2007-10-31 2007-10-31
US61/001,247 2007-10-31
PCT/US2008/082072 WO2009059195A2 (en) 2007-10-31 2008-10-31 Rationally-designed single-chain meganucleases with non-palindromic recognition sequences

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JP2015115646A Division JP6050861B2 (ja) 2007-10-31 2015-06-08 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ

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JP2011501971A JP2011501971A (ja) 2011-01-20
JP2011501971A5 JP2011501971A5 (enExample) 2011-12-22
JP5761996B2 true JP5761996B2 (ja) 2015-08-12

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JP2010532293A Active JP5761996B2 (ja) 2007-10-31 2008-10-31 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
JP2015115646A Active JP6050861B2 (ja) 2007-10-31 2015-06-08 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
JP2016193485A Pending JP2017051190A (ja) 2007-10-31 2016-09-30 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
JP2018219205A Pending JP2019062898A (ja) 2007-10-31 2018-11-22 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
JP2020196770A Pending JP2021052770A (ja) 2007-10-31 2020-11-27 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
JP2022187981A Pending JP2023029894A (ja) 2007-10-31 2022-11-25 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
JP2024187133A Pending JP2025028832A (ja) 2007-10-31 2024-10-24 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ

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JP2016193485A Pending JP2017051190A (ja) 2007-10-31 2016-09-30 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
JP2018219205A Pending JP2019062898A (ja) 2007-10-31 2018-11-22 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
JP2020196770A Pending JP2021052770A (ja) 2007-10-31 2020-11-27 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
JP2022187981A Pending JP2023029894A (ja) 2007-10-31 2022-11-25 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
JP2024187133A Pending JP2025028832A (ja) 2007-10-31 2024-10-24 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ

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US (10) US8445251B2 (enExample)
EP (4) EP3578646A3 (enExample)
JP (7) JP5761996B2 (enExample)
AU (1) AU2008318430A1 (enExample)
CA (1) CA2704383A1 (enExample)
DK (3) DK2215223T3 (enExample)
ES (3) ES2422291T3 (enExample)
WO (1) WO2009059195A2 (enExample)

Families Citing this family (131)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009095742A1 (en) 2008-01-31 2009-08-06 Cellectis New i-crei derived single-chain meganuclease and uses thereof
WO2003078619A1 (en) † 2002-03-15 2003-09-25 Cellectis Hybrid and single chain meganucleases and use thereof
EP3578646A3 (en) 2007-10-31 2020-03-18 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
CA2781693C (en) 2009-11-27 2018-12-18 Basf Plant Science Company Gmbh Chimeric endonucleases and uses thereof
AU2010325564A1 (en) 2009-11-27 2012-07-12 Basf Plant Science Company Gmbh Chimeric endonucleases and uses thereof
DE112010004582T5 (de) 2009-11-27 2012-11-29 Basf Plant Science Company Gmbh Optimierte Endonukleasen und Anwendungen davon
US20130198897A1 (en) 2010-03-22 2013-08-01 Philip Morris Products S.A. Modifying enzyme activity in plants
WO2012138901A1 (en) * 2011-04-05 2012-10-11 Cellectis Sa Method for enhancing rare-cutting endonuclease efficiency and uses thereof
DK2714936T3 (en) 2011-06-01 2019-03-25 Prec Biosciences Inc METHODS AND PRODUCTS FOR PRODUCING MANIPULATED MAMMAL CELL LINES WITH AMPLIFIED TRANSGENES
EP2612918A1 (en) 2012-01-06 2013-07-10 BASF Plant Science Company GmbH In planta recombination
WO2013166113A1 (en) 2012-05-04 2013-11-07 E. I. Du Pont De Nemours And Company Compositions and methods comprising sequences having meganuclease activity
EP3116533B1 (en) 2014-03-12 2020-08-12 Precision Biosciences, Inc. Dystrophin gene exon deletion using engineered nucleases
CN106714826A (zh) 2014-09-07 2017-05-24 西莱克塔生物科技公司 用于减弱基因表达调节抗病毒转移载体免疫应答的方法和组合物
ES2905181T3 (es) 2015-05-01 2022-04-07 Prec Biosciences Inc Deleción precisa de secuencias cromosómicas in vivo
CA2985816A1 (en) 2015-05-15 2016-11-24 The General Hospital Corporation Antagonistic anti-tumor necrosis factor receptor superfamily antibodies
EP4115894A1 (en) 2015-06-19 2023-01-11 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
JP6980534B2 (ja) 2015-06-25 2021-12-15 ザ チルドレンズ メディカル センター コーポレーション 造血幹細胞の増大、富化、および維持に関する方法および組成物
WO2017024407A1 (en) 2015-08-11 2017-02-16 The Royal Institution For The Advancement Of Learning/Mcgill University Peptidic tgf-beta antagonists
EP4530354A3 (en) 2015-09-08 2025-09-10 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
ES2768984T3 (es) 2015-10-05 2020-06-24 Prec Biosciences Inc Meganucleasas diseñadas con secuencias de reconocimiento encontradas en el gen de la región constante alfa del receptor de células T humanas
AU2016333898B2 (en) 2015-10-05 2020-11-12 Precision Biosciences, Inc. Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene
WO2017112859A1 (en) 2015-12-23 2017-06-29 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human beta-2 microglobulin gene
US11274288B2 (en) 2016-02-16 2022-03-15 Emendobio Inc. Compositions and methods for promoting homology directed repair mediated gene editing
DK3429603T3 (da) 2016-03-15 2022-03-14 Childrens Medical Center Fremgangsmåder og sammensætninger vedrørende ekspansion af hæmatopoietiske stamceller
CA3017645C (en) 2016-04-27 2021-07-13 University Of Puerto Rico 1,5-disubstituted 1,2,3-triazoles are inhibitors of rac/cdc42 gtpases
JP2019514397A (ja) 2016-05-03 2019-06-06 プレシジョン バイオサイエンシズ，インク． 血友病ａの治療に有用な操作されたヌクレアーゼ
MX2018015798A (es) 2016-06-22 2019-07-12 Univ North Carolina State Método para incrementar la eficiencia de uso de nitrógeno y/o la eficiencia de utilización de nitrógeno en plantas.
EP3487994A4 (en) * 2016-07-25 2020-01-29 Bluebird Bio, Inc. HOMING BCL11A ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE
WO2018039333A1 (en) 2016-08-23 2018-03-01 Bluebird Bio, Inc. Tim3 homing endonuclease variants, compositions, and methods of use
DK3510157T5 (da) 2016-09-08 2024-09-02 2Seventy Bio Inc Pd-1-homing-endonucleasevarianter, sammensætninger og fremgangsmåder til anvendelse
AU2017338846B2 (en) 2016-10-04 2020-09-24 Precision Biosciences, Inc. Co-stimulatory domains for use in genetically-modified cells
US10662416B2 (en) * 2016-10-14 2020-05-26 Precision Biosciences, Inc. Engineered meganucleases specific for recognition sequences in the hepatitis B virus genome
AU2017346683B2 (en) * 2016-10-17 2024-04-18 Regeneron Pharmaceuticals, Inc. TGFβR2 endonuclease variants, compositions, and methods of use
WO2018073393A2 (en) 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
JP7352807B2 (ja) 2017-04-12 2023-09-29 マジェンタ セラピューティクス インコーポレイテッド アリール炭化水素受容体アンタゴニスト及びその使用
KR20230170125A (ko) * 2017-04-21 2023-12-18 프리시젼 바이오사이언시스 인코포레이티드 Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제
WO2018201144A1 (en) 2017-04-28 2018-11-01 Precision Biosciences, Inc. Methods for reducing dna-induced cytotoxicity and enhancing gene editing in primary cells
AU2018266698A1 (en) 2017-05-08 2019-11-28 Precision Biosciences, Inc. Nucleic acid molecules encoding an engineered antigen receptor and an inhibitory nucleic acid molecule and methods of use thereof
KR102590466B1 (ko) 2017-05-25 2023-10-19 2세븐티 바이오, 인코포레이티드 Cblb 엔도뉴클레아제 변이체, 조성물 및 사용 방법
CA3067842C (en) 2017-06-23 2025-11-04 Univ Kentucky Res Found Methods of modulating the alkaloid content of a plant or part thereof
CA3068465A1 (en) 2017-06-30 2019-01-03 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
JP2020529206A (ja) 2017-07-31 2020-10-08 アール・ジエイ・レイノルズ・タバコ・カンパニー 植物におけるウイルスベースの遺伝子編集のための方法及び組成物
US20200239544A1 (en) 2017-10-03 2020-07-30 Precision Biosciences, Inc. Modified epidermal growth factor receptor peptides for use in genetically-modified cells
WO2019070974A1 (en) * 2017-10-04 2019-04-11 Bluebird Bio, Inc. PCSK9 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE
AU2018347583A1 (en) 2017-10-13 2020-05-21 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector IgM responses
EP3703715A1 (en) 2017-10-31 2020-09-09 Magenta Therapeutics, Inc. Compositions and methods for hematopoietic stem and progenitor cell transplant therapy
US20190314407A1 (en) 2017-10-31 2019-10-17 Magenta Therapeutics Inc. Compositions and methods for the expansion of hematopoietic stem and progenitor cells
WO2019089913A1 (en) 2017-11-01 2019-05-09 Precision Biosciences, Inc. Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a
JP2021505172A (ja) 2017-12-06 2021-02-18 マジェンタ セラピューティクス インコーポレイテッドＭａｇｅｎｔａ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． 造血幹細胞及び前駆細胞を動員させるための投薬レジメン
US20210002621A1 (en) * 2017-12-20 2021-01-07 Bluebird Bio, Inc. Ctla4 homing endonuclease variants, compositions, and methods of use
JP2021510150A (ja) 2018-01-03 2021-04-15 マジェンタ セラピューティクス インコーポレイテッドＭａｇｅｎｔａ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． 造血幹細胞および前駆細胞の増幅ならびに遺伝性代謝障害の処置のための組成物および方法
CN111819285B (zh) 2018-01-09 2024-08-09 希博斯美国有限公司 防碎基因和突变
CA3092372A1 (en) 2018-02-26 2019-08-29 AnTolRx, Inc. Tolerogenic liposomes and methods of use thereof
EA202092382A1 (ru) 2018-04-04 2021-02-02 САЙБАС ЮЭс ЛЛС Гены fad2 и мутации
IT201800004253A1 (it) 2018-04-05 2019-10-05 Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria.
KR102617818B1 (ko) 2018-04-12 2023-12-27 프리시젼 바이오사이언시스 인코포레이티드 인간 t 세포 수용체 알파 불변 영역 유전자에 대한 특이성을 갖는 최적화된 조작된 뉴클레아제
TW201945388A (zh) 2018-04-12 2019-12-01 美商精密生物科學公司 對b型肝炎病毒基因體中之識別序列具有特異性之最佳化之經工程化巨核酸酶
US20210230565A1 (en) * 2018-04-27 2021-07-29 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Bruton's tyrosine kinase homing endonuclease variants, compositions, and methods of use
BR112020025439A2 (pt) 2018-06-14 2021-03-16 Bluebird Bio, Inc. Receptores de antígenos quiméricos cd79a
GB201812603D0 (en) 2018-08-02 2018-09-19 British American Tobacco Investments Ltd Method
KR20210071976A (ko) 2018-09-04 2021-06-16 마젠타 테라퓨틱스 인코포레이티드 아릴 하이드로카본 수용체 길항제 및 사용 방법
WO2020072059A1 (en) * 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
GB201817971D0 (en) 2018-11-02 2018-12-19 British American Tobacco Investments Ltd Method
GB201818715D0 (en) 2018-11-16 2019-01-02 British American Tobacco Investments Ltd Method
CA3122278A1 (en) 2018-12-10 2020-06-18 Bluebird Bio, Inc. Homing endonuclease variants
US20220090047A1 (en) 2018-12-21 2022-03-24 Precision Biosciences, Inc. Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria
GB201900940D0 (en) 2019-01-23 2019-03-13 British American Tobacco Investments Ltd Method
AU2020253538A1 (en) 2019-04-03 2021-11-18 Precision Biosciences, Inc. Genetically-modified immune cells comprising a microRNA-adapted shRNA (shRNAmiR)
US20220204994A1 (en) 2019-04-05 2022-06-30 Precision Biosciences, Inc. Methods of preparing populations of genetically-modified immune cells
CA3137975A1 (en) 2019-05-07 2020-11-12 Precision Biosciences, Inc. Optimization of engineered meganucleases for recognition sequences
GB201906768D0 (en) 2019-05-14 2019-06-26 British American Tobacco Investments Ltd Method
CN114206396A (zh) 2019-05-28 2022-03-18 西莱克塔生物科技公司 用于减弱抗病毒转移载体免疫应答的方法和组合物
GB201909562D0 (en) 2019-07-03 2019-08-14 British American Tobacco Investments Ltd Method
GB201909563D0 (en) 2019-07-03 2019-08-14 British American Tobacco Investments Ltd Method
JP2022544825A (ja) 2019-08-20 2022-10-21 プレシジョン バイオサイエンシズ，インク． 細胞免疫療法のためのリンパ球枯渇投与レジメン
WO2021087305A1 (en) 2019-10-30 2021-05-06 Precision Biosciences, Inc. Cd20 chimeric antigen receptors and methods of use for immunotherapy
US20220401481A1 (en) 2019-11-01 2022-12-22 Magenta Therapeutics, Inc. Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
FI4069729T3 (fi) 2019-12-06 2025-04-01 Prec Biosciences Inc Optimoidut muokatut meganukleaasit, joilla on spesifisyys tunnistussekvenssiä varten hepatiitti b -virusgenomissa
EP3835309A1 (en) 2019-12-13 2021-06-16 KWS SAAT SE & Co. KGaA Method for increasing cold or frost tolerance in a plant
WO2021158915A1 (en) 2020-02-06 2021-08-12 Precision Biosciences, Inc. Recombinant adeno-associated virus compositions and methods for producing and using the same
WO2021205000A2 (en) 2020-04-09 2021-10-14 R.J. Reynolds Tobacco Company Method
US20230330185A1 (en) 2020-04-27 2023-10-19 Magenta Therapeutics, Inc. Methods and compositions for transducing hematopoietic stem and progenitor cells in vivo
WO2021231259A1 (en) 2020-05-11 2021-11-18 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
JP2023526310A (ja) 2020-05-12 2023-06-21 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 導入遺伝子発現のｄｒｇ特異的低減のための組成物
US20230193230A1 (en) 2020-05-12 2023-06-22 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using improved engineered meganucleases
EP4192875A1 (en) 2020-08-10 2023-06-14 Precision BioSciences, Inc. Antibodies and fragments specific for b-cell maturation antigen and uses thereof
CA3172292A1 (en) 2020-08-21 2022-02-24 Cassandra GORSUCH Engineered meganucleases having specificity for a recognition sequence in the transthyretin gene
US20230365995A1 (en) 2020-10-07 2023-11-16 Precision Biosciences, Inc. Lipid nanoparticle compositions
WO2022094180A1 (en) 2020-10-29 2022-05-05 The Trustees Of The University Of Pennsylvania Aav capsids and compositions containing same
EP4244342A1 (en) 2020-11-12 2023-09-20 Precision BioSciences, Inc. Engineered meganucleases having specificity for recognition sequences in the dystrophin gene
CA3172161A1 (en) 2021-01-08 2022-07-14 James Jefferson Smith Engineered meganucleases having specificity for a recognition sequence in the hydroxyacid oxidase 1 gene
EP4284823A1 (en) 2021-01-28 2023-12-06 Precision BioSciences, Inc. Modulation of tgf beta signaling in genetically-modified eukaryotic cells
WO2022197776A1 (en) 2021-03-16 2022-09-22 Magenta Therapeutics, Inc. Dosing regimens for hematopoietic stem cell mobilization for stem cell transplants in multiple myeloma patients
AU2022262778A1 (en) 2021-04-22 2023-11-30 Precision Biosciences, Inc. Engineered meganucleases that target human mitochondrial genomes
EP4326861B1 (en) 2021-04-22 2025-12-17 Precision Biosciences, Inc. Engineered meganucleases that target human mitochondrial genomes
WO2022226316A1 (en) 2021-04-22 2022-10-27 Precision Biosciences, Inc. Compositions and methods for generating male sterile plants
AR125467A1 (es) 2021-04-27 2023-07-19 Univ Pennsylvania Cápsides de virus adenoasociados derivados de porcinos y usos de los estos
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
WO2023070003A1 (en) 2021-10-19 2023-04-27 Precision Biosciences, Inc. Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
AU2022368911A1 (en) 2021-10-19 2024-05-30 Precision Biosciences, Inc. Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
MX2024009603A (es) 2022-02-03 2024-08-14 Nicoventures Trading Ltd Metodo.
WO2023148476A1 (en) 2022-02-03 2023-08-10 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
KR20240132133A (ko) 2022-02-04 2024-09-02 니코벤처스 트레이딩 리미티드 담배 식물들에서 알칼로이드 함량을 조절하는 방법
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
GB202205148D0 (en) 2022-04-07 2022-05-25 Nicoventures Trading Ltd Method
GB202205149D0 (en) 2022-04-07 2022-05-25 Nicoventures Trading Ltd Method
GB202205561D0 (en) 2022-04-14 2022-06-01 Nicoventures Trading Ltd Method
GB202205562D0 (en) 2022-04-14 2022-06-01 Nicoventures Trading Ltd Method
GB202206107D0 (en) 2022-04-27 2022-06-08 Nicoventures Trading Ltd Method
GB202206109D0 (en) 2022-04-27 2022-06-08 Nicoventures Trading Ltd Method
WO2023215498A2 (en) 2022-05-05 2023-11-09 Modernatx, Inc. Compositions and methods for cd28 antagonism
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
WO2024056902A2 (en) 2022-09-16 2024-03-21 Christopher Shaw Compositions and methods for treating neurological diseases
WO2024097639A1 (en) 2022-10-31 2024-05-10 Modernatx, Inc. Hsa-binding antibodies and binding proteins and uses thereof
WO2024118866A1 (en) 2022-12-01 2024-06-06 Modernatx, Inc. Gpc3-specific antibodies, binding domains, and related proteins and uses thereof
AU2023392154A1 (en) 2022-12-14 2025-06-26 King's College London Compositions and methods for treating neurological diseases
AU2024206812A1 (en) 2023-01-05 2025-08-14 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for the human t cell receptor alpha constant region gene
GB202300905D0 (en) 2023-01-20 2023-03-08 Nicoventures Trading Ltd Method
WO2024161022A2 (en) 2023-02-03 2024-08-08 King's College London Compositions and methods for treating neurological diseases
KR20250141243A (ko) 2023-02-06 2025-09-26 블루록 테라퓨틱스 엘피 데그론 융합 단백질 및 그의 생산 및 사용 방법
TW202448928A (zh) 2023-03-02 2024-12-16 美商艾羅伊治療公司 抗cd22抗體及其用途
GB202303077D0 (en) 2023-03-02 2023-04-19 Univ Oslo Brassica plants with improved seed retention
AU2024255934A1 (en) 2023-04-14 2025-11-20 Precision Biosciences, Inc. Muscle-specific expression cassettes
WO2024216116A1 (en) 2023-04-14 2024-10-17 Precision Biosciences, Inc. Muscle-specific expression cassettes
WO2024236547A1 (en) 2023-05-18 2024-11-21 Inceptor Bio, Llc Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof
WO2025046513A1 (en) 2023-08-29 2025-03-06 Inceptor Bio, Llc Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof
WO2025083169A1 (en) 2023-10-17 2025-04-24 Tenpoint Therapeutics Limited Combination of a vegf inhibitor and a complement pathway inhibitor for treating ocular disorders
WO2025101946A1 (en) 2023-11-08 2025-05-15 Precision Biosciences, Inc. Polypeptide linkers for use in engineered meganucleases
WO2025126153A2 (en) 2023-12-14 2025-06-19 Aviadobio Ltd. Compositions and methods for treating sod1-mediated neurological diseases
WO2025233894A1 (en) 2024-05-09 2025-11-13 University Of Miami Engineered meganucleases that target human mitochondrial genomes

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE122007000007I1 (de) 1986-04-09 2007-05-16 Genzyme Corp Genetisch transformierte Tiere, die ein gewünschtes Protein in Milch absondern
US4873316A (en) 1987-06-23 1989-10-10 Biogen, Inc. Isolation of exogenous recombinant proteins from the milk of transgenic mammals
US6156304A (en) 1990-12-20 2000-12-05 University Of Pittsburgh Of The Commonwealth System Of Higher Education Gene transfer for studying and treating a connective tissue of a mammalian host
US5384253A (en) 1990-12-28 1995-01-24 Dekalb Genetics Corporation Genetic transformation of maize cells by electroporation of cells pretreated with pectin degrading enzymes
US5997861A (en) 1994-10-31 1999-12-07 Burstein Laboratories, Inc. Antiviral supramolecules containing target-binding molecules and therapeutic molecules bound to spectrin
US6056973A (en) 1996-10-11 2000-05-02 Sequus Pharmaceuticals, Inc. Therapeutic liposome composition and method of preparation
US6060082A (en) 1997-04-18 2000-05-09 Massachusetts Institute Of Technology Polymerized liposomes targeted to M cells and useful for oral or mucosal drug delivery
US6511676B1 (en) 1999-11-05 2003-01-28 Teni Boulikas Therapy for human cancers using cisplatin and other drugs or genes encapsulated into liposomes
US6593308B2 (en) 1999-12-03 2003-07-15 The Regents Of The University Of California Targeted drug delivery with a hyaluronan ligand
AU2002317771A1 (en) * 2001-06-05 2002-12-16 Cellectis Methods for modifying the cpg content of polynucleotides
AU2003210996A1 (en) * 2002-02-11 2003-09-04 Zymogenetics, Inc. Materials and methods for preparing dimeric growth factors
WO2003078619A1 (en) * 2002-03-15 2003-09-25 Cellectis Hybrid and single chain meganucleases and use thereof
WO2009095742A1 (en) 2008-01-31 2009-08-06 Cellectis New i-crei derived single-chain meganuclease and uses thereof
JP2006518372A (ja) 2003-01-28 2006-08-10 セレクティス 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用
JP4095622B2 (ja) * 2004-03-31 2008-06-04 キヤノン株式会社 金結合性複合タンパク質
EP1591521A1 (en) * 2004-04-30 2005-11-02 Cellectis I-Dmo I derivatives with enhanced activity at 37 degrees C and use thereof
DK1863909T3 (da) * 2005-03-15 2010-08-30 Cellectis I-CreI-meganukleasevarianter med modificeret specificitet, fremgangsmåde til fremstilling og anvendelser deraf
WO2006097784A1 (en) 2005-03-15 2006-09-21 Cellectis I-crei meganuclease variants with modified specificity, method of preparation and uses thereof
WO2007034262A1 (en) 2005-09-19 2007-03-29 Cellectis Heterodimeric meganucleases and use thereof
EP1764414A1 (en) * 2005-09-17 2007-03-21 Icon Genetics AG Plant viral particles comprising a plurality of fusion proteins consisting of a plant viral coat protein, a peptide linker and a recombinant protein and use of such plant viral particles for protein purification
US8021867B2 (en) * 2005-10-18 2011-09-20 Duke University Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity
WO2007049095A1 (en) 2005-10-25 2007-05-03 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof
WO2007060495A1 (en) 2005-10-25 2007-05-31 Cellectis I-crei homing endonuclease variants having novel cleavage specificity and use thereof
EP1957520A4 (en) * 2005-11-29 2009-05-27 Univ Virginia Commonwealth POLYVALENTIC CHIMERIC OSPC VACCINOGEN AND DIAGNOSTIC ANTIGEN
WO2007093836A1 (en) 2006-02-13 2007-08-23 Cellectis Meganuclease variants cleaving a dna target sequence from a xp gene and uses thereof
GB0603099D0 (en) * 2006-02-16 2006-03-29 Lontra Environmental Technolog Rotary piston and cylinder devices
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
SG176487A1 (en) 2006-11-14 2011-12-29 Cellectis Meganuclease variants cleaving a dna target sequence from the hprt gene and uses thereof
WO2008059317A1 (en) 2006-11-14 2008-05-22 Cellectis Meganuclease variants cleaving a dna target sequence from the hprt gene and uses thereof
WO2008093152A1 (en) 2007-02-01 2008-08-07 Cellectis Obligate heterodimer meganucleases and uses thereof
AU2007347328B2 (en) 2007-02-19 2013-03-07 Cellectis LAGLIDADG homing endonuclease variants having novel substrate specificity and use thereof
WO2008102199A1 (en) * 2007-02-20 2008-08-28 Cellectis Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof
WO2008149176A1 (en) * 2007-06-06 2008-12-11 Cellectis Meganuclease variants cleaving a dna target sequence from the mouse rosa26 locus and uses thereof
EP2167666A2 (en) 2007-06-29 2010-03-31 Pioneer Hi-Bred International Inc. Methods for altering the genome of a monocot plant cell
EP3578646A3 (en) 2007-10-31 2020-03-18 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
WO2009074842A1 (en) 2007-12-13 2009-06-18 Cellectis Improved chimeric meganuclease enzymes and uses thereof

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