JP2006502748A - 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法 - Google Patents
遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法 Download PDFInfo
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| PCT/US2003/027958 WO2004037977A2 (en) | 2002-09-05 | 2003-09-05 | Use of chimeric nucleases to stimulate gene targeting |
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Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2015500648A (ja) * | 2011-12-16 | 2015-01-08 | ターゲットジーン バイオテクノロジーズ リミテッド | 所定の標的核酸配列を修飾するための組成物及び方法 |
Families Citing this family (358)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ITMI20030821A1 (it) * | 2003-04-18 | 2004-10-19 | Internat Ct For Genetic En Gineering And | Polipeptidi chimerici e loro uso. |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| US20080311095A1 (en) * | 2007-05-23 | 2008-12-18 | Sangamo Biosciences, Inc. | Methods and compositions for increased transgene expression |
| US11311574B2 (en) | 2003-08-08 | 2022-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for targeted cleavage and recombination |
| EP2927318B1 (en) * | 2003-08-08 | 2020-05-20 | Sangamo Therapeutics, Inc. | Methods and compositions for targeted cleavage and recombination |
| US20120196370A1 (en) | 2010-12-03 | 2012-08-02 | Fyodor Urnov | Methods and compositions for targeted genomic deletion |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| AU2005233583B2 (en) * | 2004-04-08 | 2011-02-03 | Sangamo Therapeutics, Inc. | Methods and compositions for modulating cardiac contractility |
| WO2006063031A2 (en) | 2004-12-06 | 2006-06-15 | Haplomics | Allelic variants of human factor viii |
| ATE466933T1 (de) * | 2005-03-15 | 2010-05-15 | Cellectis | I-crei-meganuklease-varianten mit modifizierter spezifität sowie verfahren zu ihrer herstellung und verwendung |
| WO2007034262A1 (en) * | 2005-09-19 | 2007-03-29 | Cellectis | Heterodimeric meganucleases and use thereof |
| EP1913149A4 (en) | 2005-07-26 | 2009-08-05 | Sangamo Biosciences Inc | TARGETED INTEGRATION AND EXPRESSION OF EXOGENOUS NUCLEIC ACID SEQUENCES |
| AU2012245168B2 (en) * | 2005-07-26 | 2014-12-11 | Sangamo Therapeutics, Inc. | Targeted Integration and Expression of Exogenous Nucleic Acid Sequences |
| DK3246403T3 (da) | 2005-10-18 | 2020-10-26 | Prec Biosciences | Rationelt designede meganucleaser med ændret sekvens-specificitet og dna-bindende affinitet |
| US20070266449A1 (en) * | 2006-05-12 | 2007-11-15 | Zivin Robert A | Generation of animal models |
| WO2007136685A2 (en) * | 2006-05-19 | 2007-11-29 | Sangamo Biosciences, Inc. | Methods and compositions for inactivation of dihydrofolate reductase |
| US7951925B2 (en) | 2006-05-25 | 2011-05-31 | Sangamo Biosciences, Inc. | Methods and compositions for gene inactivation |
| EP2027262B1 (en) * | 2006-05-25 | 2010-03-31 | Sangamo Biosciences Inc. | Variant foki cleavage half-domains |
| US20080003681A1 (en) * | 2006-06-28 | 2008-01-03 | Mahalaxmi Gita Bangera | Methods for altering cellular susceptibility to infection |
| JP2010500029A (ja) * | 2006-08-11 | 2010-01-07 | ダウ アグロサイエンシィズ エルエルシー | ジンクフィンガーヌクレアーゼ媒介相同組換え |
| WO2008060510A2 (en) | 2006-11-13 | 2008-05-22 | Sangamo Biosciences, Inc. | Zinc finger nuclease for targeting the human glucocorticoid receptor locus |
| CA2669746C (en) * | 2006-12-14 | 2017-05-16 | Dow Agrosciences Llc | Optimized non-canonical zinc finger proteins |
| EP2155868A2 (en) * | 2007-04-19 | 2010-02-24 | Codon Devices, Inc | Engineered nucleases and their uses for nucleic acid assembly |
| US8110379B2 (en) | 2007-04-26 | 2012-02-07 | Sangamo Biosciences, Inc. | Targeted integration into the PPP1R12C locus |
| AU2008275649B2 (en) | 2007-07-12 | 2013-09-05 | Sangamo Therapeutics, Inc. | Methods and compositions for inactivating alpha 1,6 fucosyltransferase (FUT 8) gene expression |
| ES2585157T3 (es) | 2007-09-27 | 2016-10-04 | Dow Agrosciences Llc | Proteínas de dedo de cinc de diseño que se dirigen a los genes de 5-enolpiruvil shikimato-3-fosfato sintasa |
| JP5763341B2 (ja) * | 2007-09-27 | 2015-08-12 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 生物学的活性のあるヌクレアーゼの迅速なインビボ同定法 |
| US8563314B2 (en) | 2007-09-27 | 2013-10-22 | Sangamo Biosciences, Inc. | Methods and compositions for modulating PD1 |
| US11235026B2 (en) | 2007-09-27 | 2022-02-01 | Sangamo Therapeutics, Inc. | Methods and compositions for modulating PD1 |
| EP2597155B1 (en) | 2007-10-25 | 2016-11-23 | Sangamo BioSciences, Inc. | Methods and compositions for targeted integration |
| WO2009076292A2 (en) * | 2007-12-07 | 2009-06-18 | Precision Biosciences, Inc. | Rationally-designed meganucleases with recognition sequences found in dnase hypersensitive regions of the human genome |
| WO2009114321A2 (en) * | 2008-03-11 | 2009-09-17 | Precision Biosciencs, Inc. | Rationally-designed meganucleases for maize genome engineering |
| US20100071083A1 (en) * | 2008-03-12 | 2010-03-18 | Smith James J | Temperature-dependent meganuclease activity |
| WO2009130695A2 (en) | 2008-04-21 | 2009-10-29 | Danziger Innovation Ltd. | Plant viral expression vectors and use of same for generating genotypic variations in plant genomes |
| JP2011519558A (ja) * | 2008-04-28 | 2011-07-14 | プレシジョン バイオサイエンシズ，インク． | 合理的に設計されたｄｎａ結合タンパク質とエフェクタードメインとの融合分子 |
| JP2011527906A (ja) | 2008-07-14 | 2011-11-10 | プレシジョン バイオサイエンシズ，インク． | Ｉ−ＣｒｅＩ由来メガヌクレアーゼの認識配列およびその使用 |
| JP5908725B2 (ja) | 2008-08-22 | 2016-04-26 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 標的一本鎖開裂および標的組込みのための方法、並びに組成物 |
| JP5756016B2 (ja) * | 2008-10-29 | 2015-07-29 | サンガモ バイオサイエンシーズ， インコーポレイテッド | グルタミンシンセターゼ遺伝子の発現を不活性化する方法及び組成物 |
| US20110023141A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved with parkinson's disease |
| US20110023150A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genome editing of genes associated with schizophrenia in animals |
| US20110023143A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of neurodevelopmental genes in animals |
| US20110023139A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in cardiovascular disease |
| US20110023140A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Rabbit genome editing with zinc finger nucleases |
| US20110016541A1 (en) * | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genome editing of sensory-related genes in animals |
| US20110016543A1 (en) * | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genomic editing of genes involved in inflammation |
| US20110030072A1 (en) * | 2008-12-04 | 2011-02-03 | Sigma-Aldrich Co. | Genome editing of immunodeficiency genes in animals |
| US20110023156A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Feline genome editing with zinc finger nucleases |
| US20110023144A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in amyotrophyic lateral sclerosis disease |
| US20110016540A1 (en) * | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genome editing of genes associated with trinucleotide repeat expansion disorders in animals |
| US20110023152A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genome editing of cognition related genes in animals |
| US20110023151A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genome editing of abc transporters |
| US20110023148A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genome editing of addiction-related genes in animals |
| US20110023146A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in secretase-associated disorders |
| US20110023158A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Bovine genome editing with zinc finger nucleases |
| US20110016546A1 (en) * | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Porcine genome editing with zinc finger nucleases |
| US20110023149A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in tumor suppression in animals |
| US20110023145A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in autism spectrum disorders |
| EP3156494B8 (en) | 2008-12-04 | 2018-09-19 | Sangamo Therapeutics, Inc. | Genome editing in rats using zinc-finger nucleases |
| US20110023153A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in alzheimer's disease |
| US20110016539A1 (en) * | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genome editing of neurotransmission-related genes in animals |
| US20110023147A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of prion disorder-related genes in animals |
| US20110023154A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Silkworm genome editing with zinc finger nucleases |
| AU2009333863B2 (en) * | 2008-12-17 | 2015-02-05 | Corteva Agriscience Llc | Targeted integration into the Zp15 locus |
| KR20100080068A (ko) * | 2008-12-31 | 2010-07-08 | 주식회사 툴젠 | 신규한 징크 핑거 뉴클레아제 및 이의 용도 |
| CA2755192C (en) * | 2009-03-20 | 2018-09-11 | Sangamo Biosciences, Inc. | Modification of cxcr4 using engineered zinc finger proteins |
| AU2010235161B2 (en) | 2009-04-09 | 2015-01-22 | Sangamo Therapeutics, Inc. | Targeted integration into stem cells |
| US8772008B2 (en) * | 2009-05-18 | 2014-07-08 | Sangamo Biosciences, Inc. | Methods and compositions for increasing nuclease activity |
| EP2449135B1 (en) | 2009-06-30 | 2016-01-06 | Sangamo BioSciences, Inc. | Rapid screening of biologically active nucleases and isolation of nuclease-modified cells |
| WO2011005849A1 (en) | 2009-07-08 | 2011-01-13 | Cellular Dynamics International, Inc. | Modified ips cells having a mutant form of human immunodeficiency virus (hiv) cellular entry gene |
| CA2769262C (en) * | 2009-07-28 | 2019-04-30 | Sangamo Biosciences, Inc. | Methods and compositions for treating trinucleotide repeat disorders |
| CN102612565B (zh) | 2009-08-11 | 2016-04-06 | 桑格摩生物科学股份有限公司 | 靶向修饰的纯合生物体 |
| US9476060B2 (en) | 2009-10-21 | 2016-10-25 | Danziger Innovations Ltd. | Generating genotypic variations in plant genomes by gamete infection |
| US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| CA2787494C (en) | 2010-01-22 | 2019-09-17 | Dow Agrosciences Llc | Targeted genomic alteration |
| JP6137596B2 (ja) | 2010-02-08 | 2017-05-31 | サンガモ セラピューティクス， インコーポレイテッド | 遺伝子操作された切断ハーフドメイン |
| US9255259B2 (en) | 2010-02-09 | 2016-02-09 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| US9567573B2 (en) | 2010-04-26 | 2017-02-14 | Sangamo Biosciences, Inc. | Genome editing of a Rosa locus using nucleases |
| AU2011256838B2 (en) | 2010-05-17 | 2014-10-09 | Sangamo Therapeutics, Inc. | Novel DNA-binding proteins and uses thereof |
| CA2805442C (en) | 2010-07-21 | 2020-05-12 | Sangamo Biosciences, Inc. | Methods and compositions for modification of an hla locus |
| WO2012012738A1 (en) * | 2010-07-23 | 2012-01-26 | Sigma-Aldrich Co., Llc | Genome editing using targeting endonucleases and single-stranded nucleic acids |
| US9057057B2 (en) * | 2010-07-27 | 2015-06-16 | The Johns Hopkins University | Obligate heterodimer variants of foki cleavage domain |
| WO2012018726A1 (en) * | 2010-08-02 | 2012-02-09 | Cellectis Sa | Method for increasing double-strand break-induced gene targeting |
| EP3511420B1 (en) | 2010-09-27 | 2021-04-07 | Sangamo Therapeutics, Inc. | Compositions for inhibiting viral entry into cells |
| EP2627665B1 (en) | 2010-10-12 | 2015-12-16 | The Children's Hospital Of Philadelphia | Methods and compositions for treating hemophilia B |
| WO2012094132A1 (en) | 2011-01-05 | 2012-07-12 | Sangamo Biosciences, Inc. | Methods and compositions for gene correction |
| BR112013030652A2 (pt) * | 2011-06-10 | 2016-12-13 | Basf Plant Science Co Gmbh | polinucleotídeo que codifica um polipeptídeo, molécula de ácido nucleico, vetor, organismo não humano, polipeptídeo e método para introduzir um ácido nucleico de interesse em um genoma de um organismo não humano |
| CA2841541C (en) | 2011-07-25 | 2019-11-12 | Sangamo Biosciences, Inc. | Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene |
| EP2758529B1 (en) | 2011-09-21 | 2019-03-20 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
| US9222105B2 (en) | 2011-10-27 | 2015-12-29 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the HPRT locus |
| WO2013074999A1 (en) | 2011-11-16 | 2013-05-23 | Sangamo Biosciences, Inc. | Modified dna-binding proteins and uses thereof |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| CA2865011C (en) | 2012-02-29 | 2021-06-15 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
| ES2683071T3 (es) | 2012-04-25 | 2018-09-24 | Regeneron Pharmaceuticals, Inc. | Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento |
| AU2013256240B2 (en) | 2012-05-02 | 2018-09-20 | Corteva Agriscience Llc | Targeted modification of malate dehydrogenase |
| CA2871524C (en) | 2012-05-07 | 2021-07-27 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated targeted integration of transgenes |
| ES2728782T3 (es) | 2012-05-25 | 2019-10-28 | Univ California | Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| WO2014011901A2 (en) | 2012-07-11 | 2014-01-16 | Sangamo Biosciences, Inc. | Methods and compositions for delivery of biologics |
| EP2890780B8 (en) | 2012-08-29 | 2020-08-19 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| BR112015004995B1 (pt) | 2012-09-07 | 2023-05-02 | Sangamo Biosciences, Inc. | Método para modificação do genoma de uma célula, uso de uma célula, semente ou planta obtida pelo referido método e nuclease de dedo de zinco sítio específica |
| UA119135C2 (uk) | 2012-09-07 | 2019-05-10 | ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі | Спосіб отримання трансгенної рослини |
| UA118090C2 (uk) | 2012-09-07 | 2018-11-26 | ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі | Спосіб інтегрування послідовності нуклеїнової кислоти, що представляє інтерес, у ген fad2 у клітині сої та специфічний для локусу fad2 білок, що зв'язується, здатний індукувати спрямований розрив |
| WO2014059173A2 (en) | 2012-10-10 | 2014-04-17 | Sangamo Biosciences, Inc. | T cell modifying compounds and uses thereof |
| CA2892448A1 (en) | 2012-12-05 | 2014-06-12 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of metabolic disorders |
| DK3363902T3 (da) | 2012-12-06 | 2020-01-02 | Sigma Aldrich Co Llc | Crispr-baseret genommodifikation og -regulering |
| US10272163B2 (en) | 2012-12-07 | 2019-04-30 | The Regents Of The University Of California | Factor VIII mutation repair and tolerance induction |
| CA2895117A1 (en) | 2012-12-13 | 2014-06-19 | James W. Bing | Precision gene targeting to a particular locus in maize |
| MX2015010841A (es) | 2013-02-20 | 2016-05-09 | Regeneron Pharma | Modificacion genetica de ratas. |
| US10227610B2 (en) | 2013-02-25 | 2019-03-12 | Sangamo Therapeutics, Inc. | Methods and compositions for enhancing nuclease-mediated gene disruption |
| ES2786253T3 (es) | 2013-03-14 | 2020-10-09 | Immusoft Corp | Métodos para la diferenciación y transducción in vitro de células b de memoria con vectores virales pseudotipados vsv-g |
| ES2749624T3 (es) | 2013-03-14 | 2020-03-23 | Caribou Biosciences Inc | Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico |
| AU2014235968B2 (en) | 2013-03-21 | 2018-05-24 | Ospedale San Raffaele Srl | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| CN105263312A (zh) | 2013-04-05 | 2016-01-20 | 美国陶氏益农公司 | 用于在植物基因组内整合外源序列的方法和组合物 |
| DK3456831T3 (da) | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
| US10604771B2 (en) | 2013-05-10 | 2020-03-31 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| EP2997146A4 (en) | 2013-05-15 | 2017-04-26 | Sangamo BioSciences, Inc. | Methods and compositions for treatment of a genetic condition |
| WO2015017866A1 (en) | 2013-08-02 | 2015-02-05 | Enevolv, Inc. | Processes and host cells for genome, pathway, and biomolecular engineering |
| CA2920899C (en) | 2013-08-28 | 2023-02-28 | Sangamo Biosciences, Inc. | Compositions for linking dna-binding domains and cleavage domains |
| US20160251410A1 (en) | 2013-09-03 | 2016-09-01 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. | Compositions and methods for expressing recombinant polypeptides |
| BR102014021800B1 (pt) | 2013-09-04 | 2022-03-29 | Dow Agrosciences Llc | Método para detectar a integração específica de sítio de uma sequência de doador de polinucleotídeo dentro de um sítio-alvo genômico |
| US10117899B2 (en) | 2013-10-17 | 2018-11-06 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells |
| EP3058072B1 (en) | 2013-10-17 | 2021-05-19 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| WO2015066643A1 (en) | 2013-11-04 | 2015-05-07 | Dow Agrosciences Llc | Optimal soybean loci |
| EP3066110B1 (en) | 2013-11-04 | 2021-12-29 | Corteva Agriscience LLC | Optimal maize loci |
| CN105980395A (zh) | 2013-11-04 | 2016-09-28 | 美国陶氏益农公司 | 最优大豆座位 |
| JP6633532B2 (ja) | 2013-11-04 | 2020-01-22 | ダウ アグロサイエンシィズ エルエルシー | 最適なトウモロコシ遺伝子座 |
| MX391904B (es) | 2013-11-11 | 2025-03-12 | Sangamo Biosciences Inc | Un represor genetico para usarse en el tratamiento de la enfermedad de huntington. |
| ES2721936T3 (es) | 2013-11-13 | 2019-08-06 | Childrens Medical Center | Regulación de la expresión genética mediada por las nucleasas |
| CA3229275A1 (en) | 2013-12-09 | 2015-06-18 | Sangamo Biosciences, Inc. | Methods and compositions for genome engineering |
| EP3460063B1 (en) | 2013-12-11 | 2024-03-13 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for the targeted modification of a genome |
| MX388127B (es) | 2013-12-11 | 2025-03-19 | Regeneron Pharma | Metodos y composiciones para la modificacion dirigida de un genoma. |
| JP2017505756A (ja) | 2013-12-13 | 2017-02-23 | ザ ジェネラル ホスピタル コーポレイション | 可溶性高分子量（ｈｍｗ）タウ種およびその用途 |
| US10774338B2 (en) | 2014-01-16 | 2020-09-15 | The Regents Of The University Of California | Generation of heritable chimeric plant traits |
| PT3102673T (pt) | 2014-02-03 | 2020-05-21 | Sangamo Biosciences Inc | Métodos e composições para tratamento de beta-talassemia |
| EP3110454B1 (en) | 2014-02-24 | 2020-11-18 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease-mediated targeted integration |
| EP3929279A1 (en) | 2014-03-18 | 2021-12-29 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of zinc finger protein expression |
| US9522936B2 (en) | 2014-04-24 | 2016-12-20 | Sangamo Biosciences, Inc. | Engineered transcription activator like effector (TALE) proteins |
| BR112016025849A2 (pt) | 2014-05-08 | 2017-10-17 | Chdi Foundation Inc | métodos e composições para o tratamento da doença de huntington |
| WO2015175642A2 (en) | 2014-05-13 | 2015-11-19 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a disease |
| US9970001B2 (en) | 2014-06-05 | 2018-05-15 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease design |
| DK3152312T3 (da) | 2014-06-06 | 2020-04-06 | Regeneron Pharma | Fremgangsmåder og sammensætninger til modifikation af et mållocus |
| US9738897B2 (en) | 2014-06-23 | 2017-08-22 | Regeneron Pharmaceuticals, Inc. | Nuclease-mediated DNA assembly |
| DK3161128T3 (en) | 2014-06-26 | 2018-11-05 | Regeneron Pharma | METHODS AND COMPOSITIONS FOR TARGETED GENTICAL MODIFICATIONS AND PROCEDURES FOR USE THEREOF |
| US20170159065A1 (en) | 2014-07-08 | 2017-06-08 | Vib Vzw | Means and methods to increase plant yield |
| WO2016005985A2 (en) | 2014-07-09 | 2016-01-14 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. | Method for reprogramming cells |
| WO2016014794A1 (en) | 2014-07-25 | 2016-01-28 | Sangamo Biosciences, Inc. | Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells |
| WO2016014837A1 (en) | 2014-07-25 | 2016-01-28 | Sangamo Biosciences, Inc. | Gene editing for hiv gene therapy |
| US9616090B2 (en) | 2014-07-30 | 2017-04-11 | Sangamo Biosciences, Inc. | Gene correction of SCID-related genes in hematopoietic stem and progenitor cells |
| IL234638A0 (en) | 2014-09-14 | 2014-12-02 | Yeda Res & Dev | NMDA receptor antagonists for the treatment of Gaucher disease |
| CN113699113A (zh) | 2014-09-16 | 2021-11-26 | 桑格摩治疗股份有限公司 | 用于造血干细胞中核酸酶介导的基因组工程化和校正的方法和组合物 |
| EP3845655B1 (en) | 2014-10-01 | 2025-01-01 | The General Hospital Corporation | Methods for increasing efficiency of nuclease-induced homology-directed repair |
| BR112017007770A2 (pt) | 2014-10-15 | 2018-01-16 | Regeneron Pharma | cultura in vitro, população de hipscs, método para modificar um locus-alvo genômico, e, hipsc. |
| GB201421096D0 (en) | 2014-11-27 | 2015-01-14 | Imp Innovations Ltd | Genome editing methods |
| CA2968808A1 (en) | 2014-11-27 | 2016-06-02 | Danziger Innovations Ltd. | Nucleic acid constructs for genome editing |
| US10889834B2 (en) | 2014-12-15 | 2021-01-12 | Sangamo Therapeutics, Inc. | Methods and compositions for enhancing targeted transgene integration |
| MX388784B (es) | 2014-12-19 | 2025-03-20 | Regeneron Pharma | Métodos y composiciones para la modificación genética dirigida a través de direccionamiento múltiple de una sola etapa. |
| US20180002379A1 (en) | 2015-01-21 | 2018-01-04 | Sangamo Therapeutics, Inc. | Methods and compositions for identification of highly specific nucleases |
| EP4218771A1 (en) | 2015-03-27 | 2023-08-02 | Yeda Research and Development Co. Ltd | Methods of treating motor neuron diseases |
| CA2981077A1 (en) | 2015-04-03 | 2016-10-06 | Dana-Farber Cancer Institute, Inc. | Composition and methods of genome editing of b-cells |
| US10179918B2 (en) | 2015-05-07 | 2019-01-15 | Sangamo Therapeutics, Inc. | Methods and compositions for increasing transgene activity |
| SG10202112057QA (en) | 2015-05-12 | 2021-12-30 | Sangamo Therapeutics Inc | Nuclease-mediated regulation of gene expression |
| US9957501B2 (en) | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
| CA2991301A1 (en) | 2015-07-13 | 2017-01-19 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| MX2017016844A (es) | 2015-07-16 | 2018-08-15 | Biokine Therapeutics Ltd | Composiciones y procedimientos para tratar cancer. |
| DK3352776T3 (da) | 2015-09-23 | 2025-06-23 | Sangamo Therapeutics Inc | Htt-repressorer og deres anvendelse |
| WO2017053729A1 (en) | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
| US10143760B2 (en) | 2015-10-28 | 2018-12-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
| EP3368673B1 (en) | 2015-10-29 | 2020-07-29 | Amyris, Inc. | Compositions and methods for production of myrcene |
| EP3380622A4 (en) | 2015-11-23 | 2019-08-07 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODIFYING IMMUNITY |
| AU2016374253B2 (en) | 2015-12-18 | 2021-10-21 | Sangamo Therapeutics, Inc. | Targeted disruption of the MHC cell receptor |
| US11352631B2 (en) | 2015-12-18 | 2022-06-07 | Sangamo Therapeutics, Inc. | Targeted disruption of the T cell receptor |
| AU2017204950A1 (en) | 2016-01-06 | 2018-08-16 | Yeda Research And Development Co. Ltd. | Compositions and methods for treating malignant, autoimmune and inflammatory diseases |
| EP3402533B1 (en) | 2016-01-15 | 2021-08-04 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| WO2017125931A1 (en) | 2016-01-21 | 2017-07-27 | The State Of Israel, Ministry Of Agriculture & Rural Development, Agricultural Research Organization (Aro) (Volcani Center) | Parthenocarpic plants and methods of producing same |
| WO2017130205A1 (en) | 2016-01-31 | 2017-08-03 | Hadasit Medical Research Services And Development Ltd. | Autosomal-identical pluripotent stem cell populations having non-identical sex chromosomal composition and uses thereof |
| JP7012650B2 (ja) | 2016-02-02 | 2022-01-28 | サンガモ セラピューティクス， インコーポレイテッド | Ｄｎａ結合ドメインと切断ドメインとを連結するための組成物 |
| WO2017138008A2 (en) | 2016-02-14 | 2017-08-17 | Yeda Research And Development Co. Ltd. | Methods of modulating protein exocytosis and uses of same in therapy |
| WO2017153982A1 (en) | 2016-03-06 | 2017-09-14 | Yeda Research And Development Co. Ltd. | Method for modulating myelination |
| CN109803530A (zh) | 2016-07-29 | 2019-05-24 | 瑞泽恩制药公司 | 包含引起c-截短的原纤维蛋白-1表达的突变的小鼠 |
| WO2018029034A1 (en) | 2016-08-09 | 2018-02-15 | Vib Vzw | Cellulose synthase inhibitors and mutant plants |
| US11774442B2 (en) | 2016-08-15 | 2023-10-03 | Enevolv, Inc. | Molecule sensor systems |
| IL247368A0 (en) | 2016-08-18 | 2016-11-30 | Yeda Res & Dev | Diagnostic and therapeutic uses of exosomes |
| WO2018039440A1 (en) | 2016-08-24 | 2018-03-01 | Sangamo Therapeutics, Inc. | Regulation of gene expression using engineered nucleases |
| IL264792B2 (en) | 2016-08-24 | 2023-10-01 | Sangamo Therapeutics Inc | Engineered target-specific zinc-finger nucleases |
| JP7256739B2 (ja) | 2016-09-07 | 2023-04-12 | サンガモ セラピューティクス， インコーポレイテッド | 肝臓遺伝子のモジュレーション |
| DK3757120T3 (da) | 2016-10-04 | 2022-07-25 | Prec Biosciences Inc | Co-stimulerende domæner til anvendelse i genetisk modificerede celler |
| KR102712926B1 (ko) | 2016-10-20 | 2024-10-07 | 상가모 테라퓨틱스, 인코포레이티드 | 파브리병의 치료를 위한 방법 및 조성물 |
| WO2018081775A1 (en) | 2016-10-31 | 2018-05-03 | Sangamo Therapeutics, Inc. | Gene correction of scid-related genes in hematopoietic stem and progenitor cells |
| CN110234765A (zh) | 2016-11-28 | 2019-09-13 | 耶达研究及发展有限公司 | 分离的多核苷酸和多肽及使用它们来表达感兴趣的表达产物的方法 |
| EP4276187A3 (en) | 2016-12-08 | 2024-01-17 | Case Western Reserve University | Methods and compositions for enhancing functional myelin production |
| US11597947B2 (en) | 2016-12-29 | 2023-03-07 | Asc Therapeutics Inc. | Gene editing method using virus |
| EP3563159A1 (en) | 2016-12-29 | 2019-11-06 | Ukko Inc. | Methods for identifying and de-epitoping allergenic polypeptides |
| CN108314736B (zh) * | 2017-01-18 | 2021-08-31 | 李燕强 | 一种促进rna降解的方法 |
| CA3049980A1 (en) | 2017-01-23 | 2018-07-26 | Regeneron Pharmaceuticals, Inc. | Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| IL250479A0 (en) | 2017-02-06 | 2017-03-30 | Sorek Rotem | Genetically engineered cells expressing a disarm system that confers resistance to phages and methods for their production |
| US20200392533A1 (en) * | 2017-04-11 | 2020-12-17 | President And Fellows Of Harvard College | In vivo gene editing of blood progenitors |
| US11820728B2 (en) | 2017-04-28 | 2023-11-21 | Acuitas Therapeutics, Inc. | Carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| JP2020518245A (ja) | 2017-05-03 | 2020-06-25 | サンガモ セラピューティクス， インコーポレイテッド | 嚢胞性線維症膜貫通コンダクタンス制御因子（ｃｆｔｒ）遺伝子の改変のための方法および組成物 |
| IL252151A0 (en) | 2017-05-07 | 2017-07-31 | Fainzilber Michael | Treatment of stress disorders |
| 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 |
| DK3635102T3 (da) | 2017-06-05 | 2025-10-27 | Regeneron Pharma | B4galt1-varianter og anvendelser deraf |
| US11512287B2 (en) | 2017-06-16 | 2022-11-29 | Sangamo Therapeutics, Inc. | Targeted disruption of T cell and/or HLA receptors |
| WO2019006464A1 (en) | 2017-06-30 | 2019-01-03 | Memorial Sloan Kettering Cancer Center | COMPOSITIONS AND METHODS OF ADOPTIVE CELLULAR THERAPY AGAINST CANCER |
| EP4775669A2 (en) | 2017-06-30 | 2026-07-15 | Precision Biosciences, Inc. | Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene |
| SG10201913147WA (en) | 2017-07-11 | 2020-02-27 | Compass Therapeutics Llc | Agonist antibodies that bind human cd137 and uses thereof |
| IL253642A0 (en) | 2017-07-24 | 2017-09-28 | Seger Rony | Combined treatment for cancer |
| KR102712142B1 (ko) | 2017-07-31 | 2024-10-07 | 리제너론 파마슈티칼스 인코포레이티드 | Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도 |
| CA3065579A1 (en) | 2017-07-31 | 2019-02-07 | Regeneron Pharmaceuticals, Inc. | Assessment of crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo |
| CN111182790A (zh) | 2017-07-31 | 2020-05-19 | 瑞泽恩制药公司 | Crispr报告体非人类动物及其用途 |
| JP2020530997A (ja) | 2017-08-08 | 2020-11-05 | サンガモ セラピューティクス， インコーポレイテッド | キメラ抗原受容体媒介性細胞標的化 |
| US11692166B2 (en) | 2017-08-23 | 2023-07-04 | Technion Research & Development Foundation Limited | Compositions and methods for improving alcohol tolerance in yeast |
| HUE073358T2 (hu) | 2017-08-29 | 2026-01-28 | Kws Saat Se & Co Kgaa | Továbbfejlesztett kék aleuron és egyéb szegregációs rendszerek |
| AU2018336161A1 (en) | 2017-09-19 | 2020-03-26 | The State Of Israel, Ministry Of Agriculture & Rural Development, Agricultural Research Organization (Aro) (Volcani Center) | Genome-edited birds |
| WO2019067875A1 (en) | 2017-09-29 | 2019-04-04 | Regeneron Pharmaceuticals, Inc. | NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS AND METHODS OF USE |
| EP3692057B9 (en) | 2017-10-03 | 2023-10-04 | Precision BioSciences, Inc. | Modified epidermal growth factor receptor peptides for use in genetically-modified cells |
| CN111565731B (zh) | 2017-10-24 | 2024-03-08 | 达莉亚·伊兰妮 | 治疗缺血性疾病的方法 |
| US11718679B2 (en) | 2017-10-31 | 2023-08-08 | Compass Therapeutics Llc | CD137 antibodies and PD-1 antagonists and uses thereof |
| EP3704238B1 (en) | 2017-11-01 | 2024-01-03 | Precision Biosciences, Inc. | Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a |
| WO2019094725A2 (en) | 2017-11-09 | 2019-05-16 | Sangamo Therapeutics, Inc. | Genetic modification of cytokine inducible sh2-containing protein (cish) gene |
| IL255664A0 (en) | 2017-11-14 | 2017-12-31 | Shachar Idit | Hematopoietic stem cells with enhanced properties |
| EP3713961A2 (en) | 2017-11-20 | 2020-09-30 | Compass Therapeutics LLC | Cd137 antibodies and tumor antigen-targeting antibodies and uses thereof |
| IL257225A (en) | 2018-01-29 | 2018-04-09 | Yeda Res & Dev | Treatment of sarcoma |
| BR112020015488A2 (pt) | 2018-02-08 | 2020-12-08 | Sangamo Therapeutics, Inc. | Nucleases alvo-específicas modificadas |
| CN112041432A (zh) | 2018-02-15 | 2020-12-04 | 纪念斯隆-凯特林癌症中心 | Foxp3靶向剂组合物以及用于过继细胞疗法的使用方法 |
| CA3093716A1 (en) | 2018-03-16 | 2019-09-19 | Immusoft Corporation | B cells genetically engineered to secrete follistatin and methods of using the same to treat follistatin-related diseases, conditions, disorders and to enhance muscle growth and strength |
| EP3592140A1 (en) | 2018-03-19 | 2020-01-15 | Regeneron Pharmaceuticals, Inc. | Transcription modulation in animals using crispr/cas systems |
| CN112585276A (zh) | 2018-04-05 | 2021-03-30 | 朱诺治疗学股份有限公司 | 产生表达重组受体的细胞的方法和相关组合物 |
| CN112585277A (zh) | 2018-04-05 | 2021-03-30 | 朱诺治疗学股份有限公司 | 表达重组受体的t细胞、相关多核苷酸和方法 |
| JP7332622B2 (ja) | 2018-04-18 | 2023-08-23 | サンガモ セラピューティクス， インコーポレイテッド | ハンチンチン（ｈｔｔ）の調節のためのジンクフィンガータンパク質組成物 |
| US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
| GB201809273D0 (en) | 2018-06-06 | 2018-07-25 | Vib Vzw | Novel mutant plant cinnamoyl-coa reductase proteins |
| AU2019297677B2 (en) | 2018-07-04 | 2024-11-07 | Ukko Inc. | Methods of de-epitoping wheat proteins and use of same for the treatment of celiac disease |
| MX2021001850A (es) | 2018-08-18 | 2021-07-15 | Harvard College | Edicion genica in situ. |
| JP7541508B2 (ja) | 2018-08-23 | 2024-08-28 | サンガモ セラピューティクス， インコーポレイテッド | 操作された標的特異的な塩基エディター |
| US11708569B2 (en) | 2018-08-29 | 2023-07-25 | University Of Copenhagen | Modified recombinant lysosomal alpha-galactosidase A and aspartylglucoaminidase having low mannose-6-phosphate and high sialic acid |
| EP4234570A3 (en) | 2018-09-18 | 2023-12-13 | Sangamo Therapeutics, Inc. | Programmed cell death 1 (pd1) specific nucleases |
| EP3852911B1 (en) | 2018-09-21 | 2025-01-22 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| CA3116576A1 (en) | 2018-10-18 | 2020-04-23 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| IL262658A (en) | 2018-10-28 | 2020-04-30 | Memorial Sloan Kettering Cancer Center | Prevention of age related clonal hematopoiesis and diseases associated therewith |
| JP7497351B2 (ja) | 2018-11-13 | 2024-06-10 | コンパス セラピューティクス リミテッド ライアビリティ カンパニー | チェックポイント分子に対する多重特異性結合性構築物およびその使用 |
| KR20210094609A (ko) | 2018-11-28 | 2021-07-29 | 포티 세븐, 인코포레이티드 | 절제 레짐에 대해 저항성인 유전적으로 변형된 hspc |
| KR20200071198A (ko) | 2018-12-10 | 2020-06-19 | 네오이뮨텍, 인코퍼레이티드 | Nrf2 발현 조절 기반 T 세포 항암면역치료법 |
| GB201820109D0 (en) | 2018-12-11 | 2019-01-23 | Vib Vzw | Plants with a lignin trait and udp-glycosyltransferase mutation |
| WO2020131632A1 (en) | 2018-12-20 | 2020-06-25 | Regeneron Pharmaceuticals, Inc. | Nuclease-mediated repeat expansion |
| EP3898661A1 (en) | 2018-12-21 | 2021-10-27 | Precision BioSciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| CN113423419A (zh) | 2019-01-04 | 2021-09-21 | 桑格摩生物治疗股份有限公司 | 用于治疗法布里病的方法和组合物 |
| PL3908568T3 (pl) | 2019-01-11 | 2024-12-02 | Acuitas Therapeutics, Inc. | Lipidy do dostarczania środków czynnych w nanocząstkach lipidowych |
| CA3106693A1 (en) | 2019-02-06 | 2020-08-13 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type i |
| MX2021010611A (es) | 2019-03-05 | 2021-11-12 | The State Of Israel Ministry Of Agriculture & Rural Development Agricultural Res Organization Aro Vo | Aves con genoma editado. |
| WO2020205838A1 (en) | 2019-04-02 | 2020-10-08 | Sangamo Therapeutics, Inc. | Methods for the treatment of beta-thalassemia |
| EP3945800A1 (en) | 2019-04-03 | 2022-02-09 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for insertion of antibody coding sequences into a safe harbor locus |
| EP4332115A3 (en) | 2019-04-03 | 2024-05-29 | Precision Biosciences, Inc. | Genetically-modified immune cells comprising a microrna-adapted shrna (shrnamir) |
| JP7506686B2 (ja) | 2019-04-04 | 2024-06-26 | 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 | ヒト化凝固因子１２遺伝子座を含む非ヒト動物 |
| AU2020253531C1 (en) | 2019-04-04 | 2022-06-16 | Regeneron Pharmaceuticals, Inc. | Methods for scarless introduction of targeted modifications into targeting vectors |
| EP3947646A1 (en) | 2019-04-05 | 2022-02-09 | Precision BioSciences, Inc. | Methods of preparing populations of genetically-modified immune cells |
| CA3136737A1 (en) | 2019-05-01 | 2020-11-05 | Juno Therapeutics, Inc. | Cells expressing a recombinant receptor from a modified tgfbr2 locus, related polynucleotides and methods |
| SG11202111372VA (en) | 2019-05-01 | 2021-11-29 | Juno Therapeutics Inc | Cells expressing a chimeric receptor from a modified cd247 locus, related polynucleotides and methods |
| WO2020247452A1 (en) | 2019-06-04 | 2020-12-10 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use |
| CN113939595A (zh) | 2019-06-07 | 2022-01-14 | 瑞泽恩制药公司 | 包括人源化白蛋白基因座的非人动物 |
| US20220259617A1 (en) | 2019-06-13 | 2022-08-18 | The General Hospital Corporation | Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells |
| KR20220024053A (ko) | 2019-06-14 | 2022-03-03 | 리제너론 파마슈티칼스 인코포레이티드 | 타우병증의 모델 |
| EP3994268A1 (en) | 2019-07-04 | 2022-05-11 | Ukko Inc. | De-epitoped alpha gliadin and use of same for the management of celiac disease and gluten sensitivity |
| IL268111A (en) | 2019-07-16 | 2021-01-31 | Fainzilber Michael | Methods of treating pain |
| US20220273715A1 (en) | 2019-07-25 | 2022-09-01 | Precision Biosciences, Inc. | Compositions and methods for sequential stacking of nucleic acid sequences into a genomic locus |
| WO2021035054A1 (en) | 2019-08-20 | 2021-02-25 | Precision Biosciences, Inc. | Lymphodepletion dosing regimens for cellular immunotherapies |
| WO2021035170A1 (en) | 2019-08-21 | 2021-02-25 | Precision Biosciences, Inc. | Compositions and methods for tcr reprogramming using fusion proteins |
| IL270306A (en) | 2019-10-30 | 2021-05-31 | Yeda Res & Dev | Prevention and treatment of pre-myeloid and myeloid malignancies |
| US20220411479A1 (en) | 2019-10-30 | 2022-12-29 | Precision Biosciences, Inc. | Cd20 chimeric antigen receptors and methods of use for immunotherapy |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| WO2021108363A1 (en) | 2019-11-25 | 2021-06-03 | Regeneron Pharmaceuticals, Inc. | Crispr/cas-mediated upregulation of humanized ttr allele |
| EP4069285A1 (en) | 2019-12-06 | 2022-10-12 | Precision BioSciences, Inc. | Methods for cancer immunotherapy, using lymphodepletion regimens and cd19, cd20 or bcma allogeneic car t cells |
| IL271656A (en) | 2019-12-22 | 2021-06-30 | Yeda Res & Dev | System and methods for identifying cells that have undergone genome editing |
| EP4096647A1 (en) | 2020-01-30 | 2022-12-07 | Yeda Research and Development Co. Ltd | Treating acute liver disease with tlr-mik inhibitors |
| WO2021158915A1 (en) | 2020-02-06 | 2021-08-12 | Precision Biosciences, Inc. | Recombinant adeno-associated virus compositions and methods for producing and using the same |
| CN116490610A (zh) * | 2020-03-04 | 2023-07-25 | 旗舰先锋创新Vi有限责任公司 | 调控基因组的方法和组合物 |
| JP7837276B2 (ja) | 2020-03-04 | 2026-03-30 | 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 | 免疫療法に対する腫瘍細胞の感作のための方法及び組成物 |
| US20230102342A1 (en) | 2020-03-23 | 2023-03-30 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use |
| US20230263121A1 (en) | 2020-03-31 | 2023-08-24 | Elo Life Systems | Modulation of endogenous mogroside pathway genes in watermelon and other cucurbits |
| WO2021224395A1 (en) | 2020-05-06 | 2021-11-11 | Cellectis S.A. | Methods for targeted insertion of exogenous sequences in cellular genomes |
| JP7891314B2 (ja) | 2020-05-06 | 2026-07-16 | セレクティス ソシエテ アノニム | 治療用タンパク質の送達のために細胞を遺伝子改変する方法 |
| WO2021231259A1 (en) | 2020-05-11 | 2021-11-18 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| KR20230022868A (ko) | 2020-05-13 | 2023-02-16 | 주노 쎄러퓨티크스 인코퍼레이티드 | 재조합 수용체를 발현하는 공여자-배치 세포의 제조 방법 |
| EP4171616A1 (en) | 2020-06-26 | 2023-05-03 | Juno Therapeutics GmbH | Engineered t cells conditionally expressing a recombinant receptor, related polynucleotides and methods |
| KR20230051172A (ko) | 2020-07-16 | 2023-04-17 | 아퀴타스 테라퓨틱스 인크. | 지질 나노 입자에 사용하기 위한 양이온성 지질 |
| BR112023001272A2 (pt) | 2020-07-24 | 2023-04-04 | Massachusetts Gen Hospital | Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células |
| EP4192875A1 (en) | 2020-08-10 | 2023-06-14 | Precision BioSciences, Inc. | Antibodies and fragments specific for b-cell maturation antigen and uses thereof |
| JP2023545999A (ja) | 2020-10-05 | 2023-11-01 | プロタリクス リミテッド | ダイサー（ｄｉｃｅｒ）様ノックアウト植物細胞 |
| US20230365995A1 (en) | 2020-10-07 | 2023-11-16 | Precision Biosciences, Inc. | Lipid nanoparticle compositions |
| CN116391040A (zh) * | 2020-10-13 | 2023-07-04 | 法国国家科学研究中心(Cnrs) | 结合接合和Crispr/Cas系统的靶向抗菌质粒及其用途 |
| WO2022079719A1 (en) | 2020-10-15 | 2022-04-21 | Yeda Research And Development Co. Ltd. | Method of treating myeloid malignancies |
| MX2023004761A (es) | 2020-10-23 | 2023-07-17 | Elo Life Systems Inc | Metodos para producir plantas de vainilla con mejor sabor y produccion agronomica. |
| JP2023549780A (ja) | 2020-11-04 | 2023-11-29 | ジュノー セラピューティクス インコーポレイテッド | 改変されたインバリアントｃｄ３免疫グロブリンスーパーファミリー鎖遺伝子座からキメラ受容体を発現する細胞ならびに関連するポリヌクレオチドおよび方法 |
| JP2023550979A (ja) | 2020-11-26 | 2023-12-06 | ウッコ インコーポレイテッド | 改変された高分子量グルテニンサブユニット及びその使用 |
| IL279559A (en) | 2020-12-17 | 2022-07-01 | Yeda Res & Dev | Controlling ubiquitination of mlkl for treatment of disease |
| WO2022165111A1 (en) | 2021-01-28 | 2022-08-04 | Precision Biosciences, Inc. | Modulation of tgf beta signaling in genetically-modified eukaryotic cells |
| CN118019546A (zh) | 2021-03-23 | 2024-05-10 | 艾欧凡斯生物治疗公司 | 肿瘤浸润淋巴细胞的cish基因编辑及其在免疫疗法中的用途 |
| WO2022226316A1 (en) | 2021-04-22 | 2022-10-27 | Precision Biosciences, Inc. | Compositions and methods for generating male sterile plants |
| WO2022236010A1 (en) * | 2021-05-06 | 2022-11-10 | The University Of North Carolina At Chapel Hill | Use of chemical epigenetic modifiers to modulate gene expression from vectors |
| AU2022272489A1 (en) | 2021-05-10 | 2023-11-30 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. | Pharmaceutical compositions for treating neurological conditions |
| EP4337769A1 (en) | 2021-05-10 | 2024-03-20 | SQZ Biotechnologies Company | Methods for delivering genome editing molecules to the nucleus or cytosol of a cell and uses thereof |
| KR20240028362A (ko) | 2021-05-28 | 2024-03-05 | 라이엘 이뮤노파마, 인크. | Nr4a3-결핍 면역 세포 및 이의 용도 |
| KR20240027676A (ko) | 2021-06-02 | 2024-03-04 | 라이엘 이뮤노파마, 인크. | Nr4a3-결핍 면역 세포 및 이의 용도 |
| WO2022264132A1 (en) | 2021-06-13 | 2022-12-22 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. | Method for reprogramming human cells |
| CA3226947A1 (en) | 2021-08-03 | 2023-02-09 | Muhammad YASSIN | Engineered tcr complex and methods of using same |
| EP4130028A1 (en) | 2021-08-03 | 2023-02-08 | Rhazes Therapeutics Ltd | Engineered tcr complex and methods of using same |
| WO2023035011A1 (en) | 2021-09-03 | 2023-03-09 | North Carolina State University | Compositions and methods for conferring resistance to geminivirus |
| WO2023062636A1 (en) | 2021-10-14 | 2023-04-20 | Weedout Ltd. | Methods of weed control |
| WO2023064872A1 (en) | 2021-10-14 | 2023-04-20 | Precision Biosciences, Inc. | Combinations of anti-bcma car t cells and gamma secretase inhibitors |
| CN118119702A (zh) | 2021-10-14 | 2024-05-31 | 隆萨销售股份有限公司 | 用于细胞外囊泡产生的经修饰的生产者细胞 |
| MX2024004812A (es) | 2021-10-19 | 2024-07-15 | Prec Biosciences Inc | Métodos de edición génica para el tratamiento de la deficiencia de alfa-1 antitripsina (aat). |
| KR20240099358A (ko) | 2021-10-27 | 2024-06-28 | 리제너론 파마슈티칼스 인코포레이티드 | B형 혈우병 치료를 위한 인자 ix를 발현하기 위한 조성물 및 방법 |
| US20250129067A1 (en) | 2021-11-08 | 2025-04-24 | Progentos Therapeutics, Inc. | Platelet-derived growth factor receptor (pdgfr) alpha inhibitors and uses thereof |
| WO2023081900A1 (en) | 2021-11-08 | 2023-05-11 | Juno Therapeutics, Inc. | Engineered t cells expressing a recombinant t cell receptor (tcr) and related systems and methods |
| US20250009800A1 (en) | 2021-11-16 | 2025-01-09 | Precision Biosciences, Inc. | Methods for cancer immunotherapy |
| AU2022408167A1 (en) | 2021-12-08 | 2024-06-06 | Regeneron Pharmaceuticals, Inc. | Mutant myocilin disease model and uses thereof |
| WO2023114943A2 (en) | 2021-12-16 | 2023-06-22 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| KR20240128067A (ko) | 2021-12-29 | 2024-08-23 | 브리스톨-마이어스 스큅 컴퍼니 | 랜딩 패드 세포주의 생성 |
| WO2023131616A1 (en) | 2022-01-05 | 2023-07-13 | Vib Vzw | Means and methods to increase abiotic stress tolerance in plants |
| WO2023131637A1 (en) | 2022-01-06 | 2023-07-13 | Vib Vzw | Improved silage grasses |
| WO2023144199A1 (en) | 2022-01-26 | 2023-08-03 | Vib Vzw | Plants having reduced levels of bitter taste metabolites |
| IL314482A (en) | 2022-02-02 | 2024-09-01 | Regeneron Pharma | Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease |
| WO2023150798A1 (en) | 2022-02-07 | 2023-08-10 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for defining optimal treatment timeframes in lysosomal disease |
| CN118613498A (zh) | 2022-02-11 | 2024-09-06 | 瑞泽恩制药公司 | 用于筛选4R Tau靶向剂的组合物和方法 |
| AU2023269134A1 (en) | 2022-05-09 | 2024-12-12 | Regeneron Pharmaceuticals, Inc. | Vectors and methods for in vivo antibody production |
| US20260083846A1 (en) | 2022-05-19 | 2026-03-26 | Lyell Immunopharma, Inc. | Polynucleotides targeting nr4a3 and uses thereof |
| KR20250022800A (ko) | 2022-06-10 | 2025-02-17 | 우모자 바이오파마 인코포레이티드 | 조작된 줄기 세포 및 그의 용도 |
| CN120659627A (zh) | 2022-07-29 | 2025-09-16 | 瑞泽恩制药公司 | 用于转铁蛋白受体（tfr）介导的脑和肌肉递送的组合物和方法 |
| EP4565603A1 (en) | 2022-08-05 | 2025-06-11 | Regeneron Pharmaceuticals, Inc. | Aggregation-resistant variants of tdp-43 |
| WO2024064958A1 (en) | 2022-09-23 | 2024-03-28 | Lyell Immunopharma, Inc. | Methods for culturing nr4a-deficient cells |
| WO2024064952A1 (en) | 2022-09-23 | 2024-03-28 | Lyell Immunopharma, Inc. | Methods for culturing nr4a-deficient cells overexpressing c-jun |
| KR20250075694A (ko) | 2022-09-28 | 2025-05-28 | 리제너론 파마슈티칼스 인코포레이티드 | 세포 기반 요법을 강화하기 위한 항체 저항성 변형 수용체 |
| WO2024077174A1 (en) | 2022-10-05 | 2024-04-11 | Lyell Immunopharma, Inc. | Methods for culturing nr4a-deficient cells |
| AU2023371614A1 (en) | 2022-11-04 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle |
| WO2024100604A1 (en) | 2022-11-09 | 2024-05-16 | Juno Therapeutics Gmbh | Methods for manufacturing engineered immune cells |
| AU2023379457A1 (en) | 2022-11-14 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes |
| US20260232729A1 (en) | 2023-02-03 | 2026-08-13 | C3S2 Gmbh | Methods for non-viral manufacturing of engineered immune cells |
| WO2024216116A1 (en) | 2023-04-14 | 2024-10-17 | Precision Biosciences, Inc. | Muscle-specific expression cassettes |
| CN121311497A (zh) | 2023-04-14 | 2026-01-09 | 精密生物科学公司 | 肌肉特异性表达盒 |
| AR133384A1 (es) | 2023-07-28 | 2025-09-24 | Regeneron Pharma | Anti-tfr:esfingomielinasa ácida para el tratamiento de la deficiencia de esfingomielinasa ácida |
| AU2024315073A1 (en) | 2023-07-28 | 2026-01-22 | Regeneron Pharmaceuticals, Inc. | Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion |
| IL326018A (en) | 2023-07-28 | 2026-03-01 | Regeneron Pharma | Introduction of anti-TFR:GAA and anti-CD63:GAA for the treatment of Pompe disease |
| EP4771049A1 (en) | 2023-08-28 | 2026-07-08 | Regeneron Pharmaceuticals, Inc. | Cxcr4 antibody-resistant modified receptors |
| IL328711A (en) | 2023-12-13 | 2026-07-01 | Umoja Biopharma Inc | Myeloid cells derived from engineered induced stem cells and methods for differentiating and using them |
| WO2025147573A2 (en) | 2024-01-05 | 2025-07-10 | Immusoft Corporation | Glp-1 expressing modified b cells for the treatment of metabolic disease |
| WO2025184567A1 (en) | 2024-03-01 | 2025-09-04 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response |
| WO2025217398A1 (en) | 2024-04-10 | 2025-10-16 | Lyell Immunopharma, Inc. | Methods for culturing cells with improved culture medium |
| WO2025235388A1 (en) | 2024-05-06 | 2025-11-13 | Regeneron Pharmaceuticals, Inc. | Transgene genomic identification by nuclease-mediated long read sequencing |
| US20250345431A1 (en) | 2024-05-10 | 2025-11-13 | Juno Therapeutics, Inc. | Genetically engineered t cells expressing a cd19 chimeric antigen receptor (car) and uses thereof for allogeneic cell therapy |
| WO2025265017A1 (en) | 2024-06-20 | 2025-12-26 | Regeneron Pharmaceuticals, Inc. | Ass1 gene insertion for the treatment of citrullinemia type i |
| WO2026009227A1 (en) | 2024-07-04 | 2026-01-08 | Yeda Research And Development Co. Ltd. | Compositions for downregulating zeb2 in macrophages and uses thereof |
| WO2026072529A1 (en) | 2024-09-24 | 2026-04-02 | University Of Florida Research Foundation, Incorporated | Mettl7a for improved embryo competence |
| WO2026083329A1 (en) | 2024-10-18 | 2026-04-23 | Precision Biosciences, Inc. | Methods of nuclease-initiated homology directed repair and replacement and compositions and uses thereof |
| WO2026083328A1 (en) | 2024-10-18 | 2026-04-23 | Precision Biosciences, Inc. | Base editing nucleotide sequences using homology directed repair |
| WO2026123001A2 (en) | 2024-12-06 | 2026-06-11 | Umoja Biopharma, Inc. | Lineage-loci engineered cell therapy and related methods of producing and using same |
| WO2026136848A2 (en) | 2024-12-20 | 2026-06-25 | Juno Therapeutics, Inc. | Cd70-targeted cars and engineered cells comprising same and related methods |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2001086989A (ja) * | 1999-09-17 | 2001-04-03 | Univ Osaka | 哺乳類型Ｃｒｅリコンビナーゼ遺伝子 |
Family Cites Families (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4942227A (en) * | 1982-01-11 | 1990-07-17 | California Institute Of Technology | Bifunctional molecules having a DNA intercalator or DNA groove binder linked to ethylene diamine tetraacetic acid, their preparation and use to cleave DNA |
| US4665184A (en) * | 1983-10-12 | 1987-05-12 | California Institute Of Technology | Bifunctional molecules having a DNA intercalator or DNA groove binder linked to ethylene diamine tetraacetic acid |
| US4795700A (en) | 1985-01-25 | 1989-01-03 | California Institute Of Technology | Nucleic acid probes and methods of using same |
| US5789155A (en) | 1987-10-30 | 1998-08-04 | California Institute Of Technology | Process for identifying nucleic acids and triple helices formed thereby |
| US5955341A (en) * | 1991-04-10 | 1999-09-21 | The Scripps Research Institute | Heterodimeric receptor libraries using phagemids |
| US5916794A (en) * | 1992-04-03 | 1999-06-29 | Johns Hopkins University | Methods for inactivating target DNA and for detecting conformational change in a nucleic acid |
| US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
| US5356802A (en) * | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
| US5487994A (en) * | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
| US5792640A (en) * | 1992-04-03 | 1998-08-11 | The Johns Hopkins University | General method to clone hybrid restriction endonucleases using lig gene |
| US5496720A (en) * | 1993-02-10 | 1996-03-05 | Susko-Parrish; Joan L. | Parthenogenic oocyte activation |
| US6331658B1 (en) * | 1993-04-20 | 2001-12-18 | Integris Baptist Medical Center, Inc. | Genetically engineered mammals for use as organ donors |
| US6242568B1 (en) * | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| US6140466A (en) * | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| CA2196419C (en) * | 1994-08-20 | 2007-08-21 | Yen Choo | Improvements in or relating to binding proteins for recognition of dna |
| US6326166B1 (en) * | 1995-12-29 | 2001-12-04 | Massachusetts Institute Of Technology | Chimeric DNA-binding proteins |
| US5789538A (en) * | 1995-02-03 | 1998-08-04 | Massachusetts Institute Of Technology | Zinc finger proteins with high affinity new DNA binding specificities |
| AU6096296A (en) | 1995-06-07 | 1996-12-30 | Ohio State University, The | Artificial restriction endonuclease |
| GB9517780D0 (en) * | 1995-08-31 | 1995-11-01 | Roslin Inst Edinburgh | Biological manipulation |
| US5801030A (en) | 1995-09-01 | 1998-09-01 | Genvec, Inc. | Methods and vectors for site-specific recombination |
| US6265196B1 (en) * | 1996-05-07 | 2001-07-24 | Johns Hopkins University | Methods for inactivating target DNA and for detecting conformational change in a nucleic acid |
| US5928914A (en) | 1996-06-14 | 1999-07-27 | Albert Einstein College Of Medicine Of Yeshiva University, A Division Of Yeshiva University | Methods and compositions for transforming cells |
| AU5195398A (en) * | 1996-10-25 | 1998-05-15 | Advanced Research And Technology Institute, Inc. | Dna sequences encoding fusions of dna repair proteins and uses thereof |
| US5945577A (en) * | 1997-01-10 | 1999-08-31 | University Of Massachusetts As Represented By Its Amherst Campus | Cloning using donor nuclei from proliferating somatic cells |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| AU757930B2 (en) | 1997-12-01 | 2003-03-13 | Roche Diagnostics Gmbh | Optimization of cells for endogenous gene activation |
| US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
| DE69942334D1 (de) * | 1998-03-02 | 2010-06-17 | Massachusetts Inst Technology | Poly-zinkfinger-proteine mit verbesserten linkern |
| JP2002522096A (ja) | 1998-08-12 | 2002-07-23 | パンジーン・コーポレイション | ドメイン特異的遺伝子の展開 |
| US6140081A (en) * | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
| BR9915191A (pt) | 1998-11-10 | 2001-12-11 | Maxygen Inc | Métodos para obter um polinucleotìdeo isolado epara produzir uma célula recombinante,protoplasto de célula vegetal, coleção deprotoplastos de célula vegetal, vegetal,polinucleotìdeo, biblioteca, um ou mais membros debiblioteca e célula recombinante alvo |
| US6503717B2 (en) | 1999-12-06 | 2003-01-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US6534261B1 (en) * | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| WO2000046385A1 (en) | 1999-02-03 | 2000-08-10 | The Children's Medical Center Corporation | Gene repair involving in vivo excision of targeting dna |
| AU2982900A (en) * | 1999-02-03 | 2000-08-25 | Children's Medical Center Corporation | Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site |
| AU4657500A (en) | 1999-04-21 | 2000-11-02 | Pangene Corporation | Locked nucleic acid hybrids and methods of use |
| US6410317B1 (en) | 1999-07-14 | 2002-06-25 | Clontech Laboratories, Inc. | Recombinase-based methods for producing expression vectors and compositions for use in practicing the same |
| US7205103B2 (en) * | 2000-02-11 | 2007-04-17 | The Salk Institute For Biological Studies | Method of regulating transcription in a cell |
| WO2001066717A2 (en) | 2000-03-03 | 2001-09-13 | The University Of Utah | Gene targeting method |
| JP2003526367A (ja) | 2000-03-16 | 2003-09-09 | ジェネティカ インコーポレイテッド | Ｒｎａ干渉の方法とｒｎａ干渉組成物 |
| US20030084471A1 (en) | 2000-03-16 | 2003-05-01 | David Beach | Methods and compositions for RNA interference |
| KR20080023768A (ko) | 2000-03-30 | 2008-03-14 | 화이트헤드 인스티튜트 포 바이오메디칼 리서치 | Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체 |
| US6492117B1 (en) | 2000-07-12 | 2002-12-10 | Gendaq Limited | Zinc finger polypeptides capable of binding DNA quadruplexes |
| US7091026B2 (en) * | 2001-02-16 | 2006-08-15 | University Of Iowa Research Foundation | Artificial endonuclease |
| ATE347593T1 (de) | 2002-01-23 | 2006-12-15 | Univ Utah Res Found | Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen |
| EP1504092B2 (en) * | 2002-03-21 | 2014-06-25 | Sangamo BioSciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
-
2003
- 2003-09-05 US US10/656,531 patent/US9447434B2/en active Active
- 2003-09-05 WO PCT/US2003/027958 patent/WO2004037977A2/en not_active Ceased
- 2003-09-05 EP EP14164933.5A patent/EP2806025B1/en not_active Expired - Lifetime
- 2003-09-05 CA CA2497913A patent/CA2497913C/en not_active Expired - Lifetime
- 2003-09-05 EP EP03796324A patent/EP1581610A4/en not_active Withdrawn
- 2003-09-05 AU AU2003298574A patent/AU2003298574B2/en not_active Expired
- 2003-09-05 JP JP2005501601A patent/JP2006502748A/ja active Pending
-
2016
- 2016-09-08 US US15/259,848 patent/US10006053B2/en not_active Expired - Lifetime
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2001086989A (ja) * | 1999-09-17 | 2001-04-03 | Univ Osaka | 哺乳類型Ｃｒｅリコンビナーゼ遺伝子 |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2015500648A (ja) * | 2011-12-16 | 2015-01-08 | ターゲットジーン バイオテクノロジーズ リミテッド | 所定の標的核酸配列を修飾するための組成物及び方法 |
| US10220052B2 (en) | 2011-12-16 | 2019-03-05 | Targetgene Biotechnologies Ltd | Compositions and methods for modifying a predetermined target nucleic acid sequence |
| US11278560B2 (en) | 2011-12-16 | 2022-03-22 | Targetgene Biotechnologies Ltd. | Compositions and methods for modifying a predetermined target nucleic acid sequence |
| US11458157B2 (en) | 2011-12-16 | 2022-10-04 | Targetgene Biotechnologies Ltd. | Compositions and methods for modifying a predetermined target nucleic acid sequence |
| US11690866B2 (en) | 2011-12-16 | 2023-07-04 | Targetgene Biotechnologies Ltd. | Compositions and methods for modifying a predetermined target nucleic acid sequence |
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| WO2004037977A3 (en) | 2005-07-28 |
| US20050026157A1 (en) | 2005-02-03 |
| AU2003298574A1 (en) | 2004-05-13 |
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