TW202027799A - 用於表現因子ix的組成物及方法 - Google Patents

用於表現因子ix的組成物及方法 Download PDF

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TW202027799A
TW202027799A TW108137786A TW108137786A TW202027799A TW 202027799 A TW202027799 A TW 202027799A TW 108137786 A TW108137786 A TW 108137786A TW 108137786 A TW108137786 A TW 108137786A TW 202027799 A TW202027799 A TW 202027799A
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sequence
factor
nucleic acid
seq
specific examples
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TW108137786A
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Chinese (zh)
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約翰 芬
漢任 黃
莫伊特利 羅伊
克迪 賴
瑞秋 薩特勒
克里斯托斯 奇拉喬斯
成 王
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美商英特利亞醫療公司
美商再生元醫藥公司
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TW108137786A 2018-10-18 2019-10-18 用於表現因子ix的組成物及方法 TW202027799A (zh)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201862747509P 2018-10-18 2018-10-18
US62/747,509 2018-10-18
US201962829009P 2019-04-03 2019-04-03
US62/829,009 2019-04-03
US201962829621P 2019-04-04 2019-04-04
US62/829,621 2019-04-04
US201962840352P 2019-04-29 2019-04-29
US62/840,352 2019-04-29

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Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL326835A (en) 2018-10-16 2026-04-01 Blueallele Llc Methods for targeted insertion of dna genes
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス，インコーポレーテッド 第ｉｘ因子を発現するための組成物及び方法
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
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
KR20240099358A (ko) 2021-10-27 2024-06-28 리제너론 파마슈티칼스 인코포레이티드 B형 혈우병 치료를 위한 인자 ix를 발현하기 위한 조성물 및 방법
EP4423271A2 (en) 2021-10-28 2024-09-04 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
IL314482A (en) 2022-02-02 2024-09-01 Regeneron Pharma Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
WO2023212677A2 (en) 2022-04-29 2023-11-02 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
AU2023269134A1 (en) 2022-05-09 2024-12-12 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2023235726A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr interference therapeutics for c9orf72 repeat expansion disease
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
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
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
KR20250075694A (ko) 2022-09-28 2025-05-28 리제너론 파마슈티칼스 인코포레이티드 세포 기반 요법을 강화하기 위한 항체 저항성 변형 수용체
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
AU2023379457A1 (en) 2022-11-14 2025-05-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
US20250002946A1 (en) 2023-06-30 2025-01-02 Regeneron Pharmaceuticals, Inc. Methods And Compositions For Increasing Homology-Directed Repair
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
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
EP4771049A1 (en) 2023-08-28 2026-07-08 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025160340A2 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Combination immunosuppression for inhibiting an immune response and enabling immunogen administration and re-administration
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
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing
WO2025265017A1 (en) 2024-06-20 2025-12-26 Regeneron Pharmaceuticals, Inc. Ass1 gene insertion for the treatment of citrullinemia type i
WO2026072529A1 (en) 2024-09-24 2026-04-02 University Of Florida Research Foundation, Incorporated Mettl7a for improved embryo competence
WO2026100298A1 (ja) * 2024-11-06 2026-05-15 学校法人自治医科大学 ポリヌクレオチド、ベクター、血友病ｂ治療用組成物、脂質ナノ粒子、細胞生成方法、及び血友病ｂ治療方法

Family Cites Families (135)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1984000560A1 (en) 1982-08-04 1984-02-16 Nat Res Dev Molecular cloning of the gene for human anti-haemophilic factor ix
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
WO1986006408A1 (en) 1985-04-22 1986-11-06 Genetics Institute, Inc. HIGH YIElD PRODUCTION OF ACTIVE FACTOR IX
US4994371A (en) 1987-08-28 1991-02-19 Davie Earl W DNA preparation of Christmas factor and use of DNA sequences
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
FR2638643B1 (fr) 1988-11-09 1991-04-12 Transgene Sa Sequence d'adn codant pour le facteur ix humain ou une proteine analogue, vecteur d'expression, cellules transformees, procede de preparation du facteur ix et produits obtenus correspondants
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
KR960702518A (ko) 1993-05-17 1996-04-27 린다 에스, 스티븐슨 Hiv 감염 및 aids에 대한 리보자임 유전자 치료(ribozyme gene therapy for hiv infection and aids)
CA2186118C (en) 1994-03-23 2010-10-19 Richard W. Hanson Compacted nucleic acids and their delivery to cells
GB9408717D0 (en) 1994-05-03 1994-06-22 Biotech & Biolog Scien Res DNA sequences
EP0760008A1 (en) 1994-05-19 1997-03-05 Dako A/S Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis
ES2355588T3 (es) 1997-03-14 2011-03-29 The Children's Hospital Of Philadelphia Composiciones para uso en terapia génica para el tratamiento de hemofilia.
WO1998054345A1 (en) 1997-05-30 1998-12-03 Baxter International Inc. Mini-adenoviral vector
WO1999003496A1 (en) 1997-07-21 1999-01-28 The University Of North Carolina At Chapel Hill Factor ix antihemophilic factor with increased clotting activity
EP1626091B1 (en) 2000-06-01 2014-07-23 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US20030143740A1 (en) 2001-10-15 2003-07-31 Christine Wooddell Processes for transposase mediated integration into mammalian cells
US20030108524A1 (en) 2001-10-18 2003-06-12 Melissa Diagana Vectors for expressing multiple transgenes
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
ES2420929T3 (es) 2002-07-18 2013-08-27 Monsanto Technology Llc Procedimientos de utilización de polinucleótidos artificiales y sus composiciones para reducir el silenciamiento de transgenes
US7579176B2 (en) 2003-10-15 2009-08-25 Newlight Technologies, Llc Method for the production of polyhydroxyalkanoic acid
ES2436741T3 (es) 2004-09-22 2014-01-07 St. Jude Children's Research Hospital Expresión mejorada de Factor IX en vectores de terapia génica
US7951925B2 (en) 2006-05-25 2011-05-31 Sangamo Biosciences, Inc. Methods and compositions for gene inactivation
EP2423307A1 (en) 2006-06-19 2012-02-29 Catalyst Biosciences, Inc. Modified coagulation factor IV polypeptides and use thereof for treatment
CN101511373B (zh) 2006-06-19 2015-12-09 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体
US8110379B2 (en) 2007-04-26 2012-02-07 Sangamo Biosciences, Inc. Targeted integration into the PPP1R12C locus
CA2720903C (en) 2008-04-14 2019-01-15 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
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
ES2985035T3 (es) 2008-09-15 2024-11-04 Uniqure Biopharma B V Mutante de polipéptido de factor IX, sus usos y su procedimiento de producción
EP3156494B8 (en) 2008-12-04 2018-09-19 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
AU2010235161B2 (en) 2009-04-09 2015-01-22 Sangamo Therapeutics, Inc. Targeted integration into stem cells
KR20120097483A (ko) 2009-07-24 2012-09-04 시그마-알드리치 컴퍼니., 엘엘씨 게놈 편집을 위한 방법
EA201290069A1 (ru) 2009-07-31 2012-07-30 Байер Хелскер Ллс Модифицированные полипептиды фактора ix и их применения
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
WO2011146885A2 (en) 2010-05-21 2011-11-24 Virxsys Corporation Compositions and methods for lentiviral expression of apoa-1 or variants thereof using spliceosome mediated rna trans-splicing
US9057057B2 (en) 2010-07-27 2015-06-16 The Johns Hopkins University Obligate heterodimer variants of foki cleavage domain
EP2627665B1 (en) 2010-10-12 2015-12-16 The Children's Hospital Of Philadelphia Methods and compositions for treating hemophilia B
ES2685611T3 (es) 2011-02-14 2018-10-10 The Children's Hospital Of Philadelphia Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo
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
WO2013120497A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
JP6342886B2 (ja) 2012-04-18 2018-06-13 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法
DK2839013T3 (da) 2012-04-18 2020-09-14 Univ Leland Stanford Junior Ikke-disruptiv-gen-targetering
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
EP4397759A3 (en) 2012-10-23 2024-10-09 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use 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
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
US10227610B2 (en) 2013-02-25 2019-03-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
ES2887254T3 (es) 2013-03-08 2021-12-22 Novartis Ag Lípidos y composiciones lipídicas para el suministro de agentes activos
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
SG11201510327TA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
CA3229275A1 (en) 2013-12-09 2015-06-18 Sangamo Biosciences, Inc. Methods and compositions for genome engineering
CN105813814B (zh) 2013-12-12 2017-09-12 3M创新有限公司 制备聚合物多层膜的方法
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
EP3110454B1 (en) 2014-02-24 2020-11-18 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration
WO2015200555A2 (en) 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Rna modification to engineer cas9 activity
EP3169776B1 (en) 2014-07-14 2025-09-03 The Regents of The University of California Crispr/cas transcriptional modulation
CN106794141B (zh) 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
CN113337533A (zh) 2014-12-23 2021-09-03 先正达参股股份有限公司 用于鉴定和富集包含位点特异性基因组修饰的细胞的方法和组合物
AU2016226077B2 (en) 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
JP6851319B2 (ja) 2015-04-27 2021-03-31 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 ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
EP4378487A3 (en) 2015-06-23 2024-09-04 The Children's Hospital of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
US11279928B2 (en) 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
CA2991301A1 (en) 2015-07-13 2017-01-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
EP3159407A1 (en) 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
US10143760B2 (en) 2015-10-28 2018-12-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
US11866727B2 (en) * 2015-11-06 2024-01-09 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1A
CN106893739A (zh) 2015-11-17 2017-06-27 香港中文大学 用于靶向基因操作的新方法和系统
EP3380622A4 (en) 2015-11-23 2019-08-07 Sangamo Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODIFYING IMMUNITY
WO2017093804A2 (en) * 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
EP3392337B1 (en) 2015-12-18 2024-03-06 Japan Science and Technology Agency Genetic modification non-human organism, egg cells, fertilized eggs, and method for modifying target genes
EP3402533B1 (en) 2016-01-15 2021-08-04 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
WO2017136794A1 (en) 2016-02-03 2017-08-10 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
US11083799B2 (en) 2016-03-16 2021-08-10 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
ES3030658T3 (en) 2016-03-30 2025-07-01 Intellia Therapeutics Inc Lipid nanoparticle formulations for crispr/cas components
CA3026110A1 (en) 2016-04-19 2017-11-02 The Broad Institute, Inc. Novel crispr enzymes and systems
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
CN105950626B (zh) 2016-06-17 2018-09-28 新疆畜牧科学院生物技术研究所 基于CRISPR/Cas9获得不同毛色绵羊的方法及靶向ASIP基因的sgRNA
CA3030587A1 (en) 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
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
WO2018058064A1 (en) 2016-09-23 2018-03-29 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing
KR102712926B1 (ko) 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
EP3551757A1 (en) 2016-12-08 2019-10-16 Intellia Therapeutics, Inc. Modified guide rnas
US20180185516A1 (en) 2016-12-09 2018-07-05 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
JP7383478B2 (ja) 2016-12-22 2023-11-20 インテリア セラピューティクス，インコーポレイテッド アルファ１アンチトリプシン欠乏症を治療するための組成物および方法
US11597947B2 (en) 2016-12-29 2023-03-07 Asc Therapeutics Inc. Gene editing method using virus
US11938197B2 (en) 2017-01-10 2024-03-26 The Sydney Children's Hospitals Network (Randwick And Westmead (Incorporating The Royal Alexandra Hospital For Children) Polynucleotides and vectors for the expression of transgenes
JP7215716B2 (ja) 2017-01-13 2023-01-31 学校法人自治医科大学 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのａａｖベクター
IL268110B2 (en) 2017-01-26 2024-01-01 Sangamo Therapeutics Inc B-cell engineering
EP3585899A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders
JP2020519272A (ja) 2017-05-09 2020-07-02 エモリー ユニバーシティー 凝固因子変異体及びその使用
TWI753168B (zh) 2017-05-22 2022-01-21 日商武田藥品工業股份有限公司 用於血友病ｂ基因療法之編碼具有增強表現的重組ｆｉｘ變異體之病毒載體
US11667934B2 (en) 2017-06-15 2023-06-06 Toolgen Incorporated Platform for expressing protein of interest in liver
CN111032867A (zh) 2017-06-16 2020-04-17 应用干细胞有限公司 具有提高的敲入效率的基因编辑方法
GB2566453B (en) 2017-09-12 2021-01-13 Univ Of Northumbria At Newcastle Impactor for aerosol component collection
EP3688162B1 (en) 2017-09-29 2024-03-06 Intellia Therapeutics, Inc. Formulations
IL311278B2 (en) 2017-09-29 2026-05-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing
US20220080055A9 (en) 2017-10-17 2022-03-17 Crispr Therapeutics Ag Compositions and methods for gene editing for hemophilia a
EP3704245A1 (en) 2017-11-01 2020-09-09 Novartis AG Synthetic rnas and methods of use
EP3714055A1 (en) * 2017-11-21 2020-09-30 CRISPR Therapeutics AG Materials and methods for treatment of autosomal dominant retinitis pigmentosa
US11891635B2 (en) 2017-12-21 2024-02-06 Max-Delbrück-Centrum Für Molekulare Medizin In Der Helmholtz-Gemeinschaft Nucleic acid sequence replacement by NHEJ
EP3787694A4 (en) 2018-04-29 2022-05-18 University of Massachusetts RAAV-MEDIATED NUCLEASE-ASSOCIATED VECTOR INTEGRATION (RAAV-NAVI)
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
JP7832761B2 (ja) 2018-06-08 2026-03-18 インテリア セラピューティクス，インコーポレイテッド 遺伝子編集のための修飾されたガイドｒｎａ
WO2019239361A1 (en) 2018-06-14 2019-12-19 Novartis Ag Method for sequence insertion using crispr
GB2596224B (en) 2018-06-19 2022-09-07 Univ Texas Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids
EP3814499A2 (en) 2018-06-28 2021-05-05 CRISPR Therapeutics AG Compositions and methods for genomic editing by insertion of donor polynucleotides
WO2020032986A1 (en) * 2018-08-10 2020-02-13 Logicbio Therapeutics, Inc. Non-disruptive gene therapy for the treatment of mma
IL326835A (en) 2018-10-16 2026-04-01 Blueallele Llc Methods for targeted insertion of dna genes
BR112021007343A2 (pt) 2018-10-18 2021-08-03 Intellia Therapeutics, Inc. composições e métodos para expressão de transgene a partir de um lócus de albumina
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス，インコーポレーテッド 第ｉｘ因子を発現するための組成物及び方法
MX2021004276A (es) 2018-10-18 2021-09-08 Intellia Therapeutics Inc Composiciones y metodos para tratar deficiencia de alfa-1 antitripsina.
CN113272428A (zh) 2018-10-18 2021-08-17 英特利亚治疗股份有限公司 核酸构建体和使用方法

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