ES2575412T3 - Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas - Google Patents

Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas Download PDF

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ES2575412T3
ES2575412T3 ES13165733.0T ES13165733T ES2575412T3 ES 2575412 T3 ES2575412 T3 ES 2575412T3 ES 13165733 T ES13165733 T ES 13165733T ES 2575412 T3 ES2575412 T3 ES 2575412T3
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James Jefferson Smith
Derek Jantz
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Precision Biosciences Inc
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Precision Biosciences Inc
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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    • C12Y301/04Phosphoric diester hydrolases (3.1.4)

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
ES13165733.0T 2007-10-31 2008-10-31 Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas Active ES2575412T3 (es)

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US1247P 1996-02-28
US124707P 2007-10-31 2007-10-31

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ES13165733.0T Active ES2575412T3 (es) 2007-10-31 2008-10-31 Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas
ES16163925T Active ES2732735T3 (es) 2007-10-31 2008-10-31 Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas
ES08845549T Active ES2422291T3 (es) 2007-10-31 2008-10-31 Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas

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ES08845549T Active ES2422291T3 (es) 2007-10-31 2008-10-31 Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas

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US (10) US8445251B2 (cg-RX-API-DMAC7.html)
EP (4) EP2660317B1 (cg-RX-API-DMAC7.html)
JP (7) JP5761996B2 (cg-RX-API-DMAC7.html)
AU (1) AU2008318430A1 (cg-RX-API-DMAC7.html)
CA (1) CA2704383A1 (cg-RX-API-DMAC7.html)
DK (3) DK3098309T3 (cg-RX-API-DMAC7.html)
ES (3) ES2575412T3 (cg-RX-API-DMAC7.html)
WO (1) WO2009059195A2 (cg-RX-API-DMAC7.html)

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
EP2660317B1 (en) 2007-10-31 2016-04-06 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
US9404099B2 (en) 2009-11-27 2016-08-02 Basf Plant Science Company Gmbh Optimized endonucleases and uses thereof
JP2013511979A (ja) 2009-11-27 2013-04-11 ビーエーエスエフ プラント サイエンス カンパニー ゲーエムベーハー キメラエンドヌクレアーゼおよびその使用
EP2504429B1 (en) 2009-11-27 2016-04-20 BASF Plant Science Company GmbH Chimeric endonucleases and uses thereof
WO2011117249A1 (en) 2010-03-22 2011-09-29 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
DK3489366T3 (da) 2011-06-01 2020-02-24 Prec Biosciences Inc Fremgangsmåder og produkter til produktion af manipulerede mammale cellelinier med forstærkede transgener
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
US20170106055A1 (en) 2014-03-12 2017-04-20 Precision Biosciences, Inc. Dystrophin Gene Exon Deletion Using Engineered Nucleases
IL292574A (en) 2014-09-07 2022-06-01 Selecta Biosciences Inc Methods and compositions for attenuating anti-viral transfer vector immune responses
EP4015633A1 (en) 2015-05-01 2022-06-22 Precision Biosciences, Inc. Precise deletion of chromosomal sequences in vivo and treatment of nucleotide repeat expansion disorders using engineered nucleases
FI3447075T3 (fi) 2015-05-15 2023-11-07 Massachusetts Gen Hospital Antagonistisia anti-tuumorinekroositekijäreseptorin superperheen vasta-aineita
WO2016205825A1 (en) 2015-06-19 2016-12-22 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
EP3313420B1 (en) 2015-06-25 2024-03-13 The Children's Medical Center Corporation Methods and compositions relating to hematopoietic stem cell expansion, enrichment, and maintenance
EP3334750B1 (en) 2015-08-11 2022-10-05 Anie Philip Peptidic tgf-beta antagonists
AU2016321244B2 (en) 2015-09-08 2020-09-24 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
KR20250013291A (ko) 2015-10-05 2025-01-31 프리시젼 바이오사이언시스 인코포레이티드 변형된 인간 t 세포 수용체 알파 불변 영역 유전자를 포함하는 유전자 변형된 세포
EP3940070A1 (en) * 2015-10-05 2022-01-19 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene
ES2901000T3 (es) 2015-12-23 2022-03-21 Prec Biosciences Inc Meganucleasas de ingeniería con secuencias de reconocimiento encontradas en el gen de la microglobulina beta-2 humana
US11274288B2 (en) 2016-02-16 2022-03-15 Emendobio Inc. Compositions and methods for promoting homology directed repair mediated gene editing
AU2017235461B2 (en) 2016-03-15 2023-02-23 Children's Medical Center Corporation Methods and compositions relating to hematopoietic stem cell expansion
GB2570382B (en) 2016-04-27 2020-07-29 Univ Puerto Rico 1,5-disubstituted 1,2,3-triazoles are inhibitors of Rac/Cdc42 GTPases
DK3452583T3 (da) 2016-05-03 2022-01-10 Prec Biosciences Inc Konstruerede nukleaser, der er nyttige til behandling af hæmofili a
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.
AU2017301609A1 (en) * 2016-07-25 2019-02-21 Bluebird Bio, Inc. BCL11A homing endonuclease variants, compositions, and methods of use
EP3504335A4 (en) 2016-08-23 2020-04-15 Bluebird Bio, Inc. HOMING TIM3 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
ES2811500T3 (es) 2016-10-04 2021-03-12 Prec Biosciences Inc Dominios coestimuladores para su uso en células genéticamente modificadas
PE20191353A1 (es) 2016-10-14 2019-10-01 Prec Biosciences Inc Meganucleasas disenadas especificamente para el reconocimiento de secuencias en el genoma del virus de la hepatitis b
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
IL293956A (en) 2017-04-12 2022-08-01 Magenta Therapeutics Inc Aryl hydrocarbon receptor antagonist and its uses
ES2953153T3 (es) 2017-04-21 2023-11-08 Prec Biosciences Inc Meganucleasas diseñadas específicas para secuencias de reconocimiento en el gen pcsk9
EP3615662A1 (en) 2017-04-28 2020-03-04 Precision Biosciences, Inc. Methods for reducing dna-induced cytotoxicity and enhancing gene editing in primary cells
JP7170666B2 (ja) 2017-05-08 2022-11-14 プレシジョン バイオサイエンシズ,インク. 操作された抗原受容体をコードする核酸分子及び阻害性核酸分子、並びにそれらの使用方法
JP7191042B2 (ja) 2017-05-25 2022-12-16 2セブンティ バイオ インコーポレイテッド Cblbエンドヌクレアーゼバリアント、組成物、および使用方法
MX2019015413A (es) 2017-06-23 2020-07-20 Univ Kentucky Res Found Metodo.
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
EP3692057B9 (en) 2017-10-03 2023-10-04 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
KR20200086670A (ko) 2017-10-13 2020-07-17 셀렉타 바이오사이언시즈, 인크. 항바이러스 전달 벡터 IgM 반응을 약화시키기 위한 방법 및 조성물
US20190343885A1 (en) 2017-10-31 2019-11-14 Magenta Therapeutics Inc. Compositions and methods for hematopoietic stem and progenitor cell transplant therapy
CA3079405A1 (en) 2017-10-31 2019-05-09 Magenta Therapeutics Inc. Compositions and methods for the expansion of hematopoietic stem and progenitor cells
US20200299658A1 (en) 2017-11-01 2020-09-24 Precision Biosciences, Inc. Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a
SG11202004913TA (en) 2017-12-06 2020-06-29 Magenta Therapeutics Inc Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
US20210002621A1 (en) * 2017-12-20 2021-01-07 Bluebird Bio, Inc. Ctla4 homing endonuclease variants, compositions, and methods of use
US20200338132A1 (en) 2018-01-03 2020-10-29 Magenta Therapeutics Inc. Compositions and methods for the expansion of hematopoietic stem and progenitor cells and treatment of inherited metabolic disorders
US11359208B2 (en) 2018-01-09 2022-06-14 Cibus Us Llc Shatterproof genes and mutations
KR20200139679A (ko) 2018-02-26 2020-12-14 안톨알엑스, 인크. 면역관용성 리포솜 및 그의 사용 방법
AU2019247403B2 (en) 2018-04-04 2025-04-10 Cibus Europe, B.V. FAD2 genes and mutations
IT201800004253A1 (it) 2018-04-05 2019-10-05 Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria.
IL277883B2 (en) 2018-04-12 2023-12-01 Prec Biosciences Inc Optimized engineered nucleases having specificity for the human t cell receptor alpha constant region gene
US11142750B2 (en) 2018-04-12 2021-10-12 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
EP3784790A4 (en) * 2018-04-27 2022-02-09 Seattle Children's Hospital d/b/a Seattle Children's Research Institute BRUTON TYROSINE KINASE HOMING ENDONUCLEAR VARIANTS, COMPOSITIONS AND METHOD OF USE
AU2019287720B2 (en) 2018-06-14 2025-08-28 Regeneron Pharmaceuticals, Inc. CD79A chimeric antigen receptors
GB201812603D0 (en) 2018-08-02 2018-09-19 British American Tobacco Investments Ltd Method
WO2020051207A2 (en) 2018-09-04 2020-03-12 Magenta Therapeutics Inc. Aryl hydrocarbon receptor antagonists and methods of use
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
US12404500B2 (en) 2018-12-10 2025-09-02 Novo Nordisk A/S 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
KR20240122580A (ko) 2019-04-03 2024-08-12 프리시젼 바이오사이언시스 인코포레이티드 마이크로RNA-적응 shRNA(shRNAmiR)를 포함하는 유전자-변형 면역 세포
CA3136265A1 (en) 2019-04-05 2020-10-08 Precision Biosciences, Inc. Methods of preparing populations of genetically-modified immune cells
CN120843482A (zh) 2019-05-07 2025-10-28 精密生物科学公司 用于识别序列的工程化大范围核酸酶的优化
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
CA3148179A1 (en) 2019-08-20 2021-02-25 Bruce J. Mccreedy Jr. Lymphodepletion dosing regimens for cellular immunotherapies
US20220411479A1 (en) 2019-10-30 2022-12-29 Precision Biosciences, Inc. Cd20 chimeric antigen receptors and methods of use for immunotherapy
WO2021087406A1 (en) 2019-11-01 2021-05-06 Magenta Therapeutics, Inc. Dosing regimens for the mobilization of hematopoietic stem and progentor cells
DK4069729T3 (da) 2019-12-06 2025-04-07 Prec Biosciences Inc Optimerede, modificerede meganukleaser med specificitet for en genkendelsessekvens i hepatitis b-virusgenomet
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
KR20220165764A (ko) 2020-04-09 2022-12-15 아아르. 제이. 레날드즈 토바코 캄파니 니코티아나 타바쿰에서 니코틴 수준을 조절하는 방법
CN115997009A (zh) 2020-04-27 2023-04-21 美真达治疗公司 用于在体内转导造血干细胞和祖细胞的方法和组合物
WO2021231259A1 (en) 2020-05-11 2021-11-18 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
EP4150068A1 (en) 2020-05-12 2023-03-22 Precision BioSciences, Inc. Treatment of retinitis pigmentosa using improved engineered meganucleases
WO2022035793A1 (en) 2020-08-10 2022-02-17 Precision Biosciences, Inc. Antibodies and fragments specific for b-cell maturation antigen and uses thereof
US20240011003A1 (en) 2020-08-21 2024-01-11 Precision Biosciences, Inc. 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
JP2023550581A (ja) 2020-10-29 2023-12-04 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 Aavカプシド及びそれを含有する組成物
CA3172481A1 (en) 2020-11-12 2022-05-19 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
US20250127811A1 (en) 2021-01-28 2025-04-24 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
WO2022226316A1 (en) 2021-04-22 2022-10-27 Precision Biosciences, Inc. Compositions and methods for generating male sterile plants
CA3173245A1 (en) 2021-04-22 2022-10-22 James Jefferson Smith Engineered meganucleases that target human mitochondrial genomes
AR125467A1 (es) 2021-04-27 2023-07-19 Univ Pennsylvania Cápsides de virus adenoasociados derivados de porcinos y usos de los estos
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
EP4419660A2 (en) 2021-10-19 2024-08-28 Precision BioSciences, Inc. Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
EP4419111A1 (en) 2021-10-19 2024-08-28 Precision Biosciences, Inc. Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
AU2023214690A1 (en) 2022-02-03 2024-08-15 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
AU2023215810A1 (en) 2022-02-03 2024-08-15 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
MX2024009605A (es) 2022-02-04 2024-08-14 Nicoventures Trading Ltd Metodo para modular el contenido de alcaloides en plantas de tabaco.
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
GB202205149D0 (en) 2022-04-07 2022-05-25 Nicoventures Trading Ltd Method
GB202205148D0 (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
GB202206109D0 (en) 2022-04-27 2022-06-08 Nicoventures Trading Ltd Method
GB202206107D0 (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
WO2024126696A1 (en) 2022-12-14 2024-06-20 King's College London Compositions and methods for treating neurological diseases
EP4646223A1 (en) 2023-01-05 2025-11-12 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
CN120641566A (zh) 2023-02-06 2025-09-12 蓝岩治疗有限公司 降解决定子融合蛋白及其产生方法和用途
GB202303077D0 (en) 2023-03-02 2023-04-19 Univ Oslo Brassica plants with improved seed retention
IL322995A (en) 2023-03-02 2025-10-01 Alloy Therapeutics Inc Anti-CD22 antibodies and their uses
WO2024216118A1 (en) 2023-04-14 2024-10-17 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
JP2005523001A (ja) * 2002-02-11 2005-08-04 ザイモジェネティクス,インコーポレイティド 二量体増殖因子を調製するための材料と方法
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
US20060153826A1 (en) * 2003-01-28 2006-07-13 Sylvain Arnould Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof
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
WO2006097784A1 (en) 2005-03-15 2006-09-21 Cellectis I-crei meganuclease variants with modified specificity, method of preparation and uses thereof
EP2325307A1 (en) 2005-03-15 2011-05-25 Cellectis I-crel 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
ES2533370T3 (es) 2005-10-18 2015-04-09 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
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
AU2006320246A1 (en) * 2005-11-29 2007-06-07 Virginia Commonwealth University Polyvalent 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
JP5453097B2 (ja) 2006-11-14 2014-03-26 セレクティス Hprt遺伝子からのdna標的配列を切断するメガヌクレアーゼ変異型及びその使用
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
US20100086533A1 (en) 2007-02-19 2010-04-08 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
EP2568048A1 (en) 2007-06-29 2013-03-13 Pioneer Hi-Bred International, Inc. Methods for altering the genome of a monocot plant cell
EP2660317B1 (en) 2007-10-31 2016-04-06 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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