CN115141836B - 肝特异性构建体、因子viii表达盒及其使用方法 - Google Patents

肝特异性构建体、因子viii表达盒及其使用方法 Download PDF

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CN115141836B
CN115141836B CN202210744050.5A CN202210744050A CN115141836B CN 115141836 B CN115141836 B CN 115141836B CN 202210744050 A CN202210744050 A CN 202210744050A CN 115141836 B CN115141836 B CN 115141836B
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hfviii
sequence
expression
protein
fviii
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CN115141836A (zh
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B·E·赖利
G·K·李
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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CN202210744050.5A 2015-10-28 2016-07-13 肝特异性构建体、因子viii表达盒及其使用方法 Active CN115141836B (zh)

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US201562247469P 2015-10-28 2015-10-28
US62/247,469 2015-10-28
US201662307897P 2016-03-14 2016-03-14
US62/307,897 2016-03-14
US201662315438P 2016-03-30 2016-03-30
US62/315,438 2016-03-30
US201662326229P 2016-04-22 2016-04-22
US62/326,229 2016-04-22
US201662355106P 2016-06-27 2016-06-27
US62/355,106 2016-06-27
CN202210744050.5A CN115141836B (zh) 2015-10-28 2016-07-13 肝特异性构建体、因子viii表达盒及其使用方法
PCT/US2016/042099 WO2017074526A1 (en) 2015-10-28 2016-07-13 Liver-specific constructs, factor viii expression cassettes and methods of use thereof
CN201680067341.8A CN108473976B (zh) 2015-10-28 2016-07-13 肝特异性构建体、因子viii表达盒及其使用方法

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AU (1) AU2016343887B2 (https=)
CA (1) CA3002524A1 (https=)
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Families Citing this family (89)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
EP3322297B1 (en) 2015-07-13 2024-12-04 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
CA3002524A1 (en) * 2015-10-28 2017-05-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof
PE20231949A1 (es) 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
PE20190401A1 (es) 2016-07-26 2019-03-13 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018049009A2 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
CA3039673A1 (en) 2016-10-20 2018-04-26 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
CN110214184B (zh) 2016-12-01 2024-07-02 桑格摩生物治疗股份有限公司 τ蛋白调节剂以及用于其递送的方法和组合物
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510038A (ja) 2017-03-09 2020-04-02 プレジデント アンド フェローズ オブ ハーバード カレッジ がんワクチン
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
AU2018312565A1 (en) * 2017-08-01 2020-02-27 Spark Therapeutics, Inc. Factor VIII (FVIII) gene therapy methods
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US20190247517A1 (en) * 2017-10-17 2019-08-15 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing for hemophilia a
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CA3088180A1 (en) 2018-01-12 2019-07-18 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting transferrin
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN111902164A (zh) 2018-02-01 2020-11-06 同源药物公司 用于恢复pah基因功能的腺相关病毒组合物及其使用方法
CA3089587A1 (en) 2018-02-08 2019-08-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases
WO2019161310A1 (en) * 2018-02-16 2019-08-22 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing by targeting fibrinogen-alpha
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
BR112020022722A8 (pt) 2018-05-09 2022-01-18 Biomarin Pharm Inc Genoma e seu uso, vetor, partícula, molécula de ácido nucleico isolada, ácido nucleico, composição, tratamento, método
US20200069819A1 (en) * 2018-05-14 2020-03-05 Biomarin Pharmaceutical Inc. Stable expression of aav vectors in juvenile subjects
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
EP3810782A2 (en) * 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020028830A1 (en) 2018-08-03 2020-02-06 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
US12290574B2 (en) 2018-08-24 2025-05-06 Spark Therapeutics, Inc. Optimized promoter sequences, intron-free expression constructs and methods of use
IL281615B2 (en) 2018-09-21 2026-01-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
EP3861131A4 (en) * 2018-10-01 2022-06-15 Ultragenyx Pharmaceutical Inc. GENE THERAPY FOR THE TREATMENT OF PROPIONIC ACIDosis
KR20210070344A (ko) 2018-10-02 2021-06-14 상가모 테라퓨틱스, 인코포레이티드 타우 단백질의 조정을 위한 방법 및 조성물
JP7460643B2 (ja) * 2018-10-16 2024-04-02 ブルーアレル， エルエルシー 遺伝子へのｄｎａの標的化挿入のための方法
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
JP7543259B2 (ja) 2018-10-18 2024-09-02 アクイタス セラピューティクス インコーポレイテッド 活性剤の脂質ナノ粒子送達のための脂質
US20210403948A1 (en) * 2018-10-26 2021-12-30 Vrije Universiteit Brussel Liver-Specific Nucleic Acid Regulatory Elements and Methods and Use Thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
MY202837A (en) 2019-01-11 2024-05-24 Acuitas Therapeutics Inc Lipids for lipid nanoparticle delivery of active agents
GB201900741D0 (en) 2019-01-18 2019-03-06 Synpromics Ltd Liver-specifc inducible prometers and methods of use thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3826664A4 (en) 2019-02-06 2022-10-26 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type i
US11529427B2 (en) 2019-02-15 2022-12-20 Crispr Therapeutics Ag Gene editing for hemophilia A with improved factor VIII expression
WO2020186207A2 (en) 2019-03-13 2020-09-17 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing fviii therapeutics
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
ES2966625T3 (es) * 2019-04-04 2024-04-23 Regeneron Pharma Roedores que comprenden un locus del factor de coagulación 12 humanizado
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN110229849B (zh) * 2019-05-31 2021-09-17 中国科学院深圳先进技术研究院 时空调控型uPA基因表达非病毒载体及其制备方法和应用
US11622547B2 (en) 2019-06-07 2023-04-11 Regeneran Pharmaceuticals, Inc. Genetically modified mouse that expresses human albumin
CN110684798A (zh) * 2019-09-03 2020-01-14 深圳新诺微环生物科技有限公司 肌肉靶向的微环dna基因治疗
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN113058041B (zh) * 2020-08-27 2022-04-05 华东师范大学 一种用于治疗庞贝氏病的产品
CN113088519B (zh) * 2019-12-23 2024-02-02 苏州华毅乐健生物科技有限公司 一种分离的核酸分子及其应用
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
IL294713A (en) * 2020-01-16 2022-09-01 Takeda Pharmaceuticals Co Adeno associated virus based gene therapy for phenylketonuria
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
WO2022016070A1 (en) 2020-07-16 2022-01-20 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
JP2023548816A (ja) 2020-11-03 2023-11-21 ファイザー・インク 陰イオン交換クロマトグラフィーによるａａｖベクターの精製方法
KR102536321B1 (ko) 2020-12-09 2023-05-26 재단법인 아산사회복지재단 인체 유래 유전자 발현 유도 모듈에 기반한 간 특이 유전자 발현 시스템
US20240066146A1 (en) 2020-12-18 2024-02-29 Sangamo Therapeutics, Inc. Improved pharmaceutical compositions containing adeno-associated viral vector
JP2024503234A (ja) 2020-12-23 2024-01-25 ファイザー・インク 親和性クロマトグラフィーによるａａｖベクターの精製方法
MX2023015329A (es) 2021-06-22 2024-01-23 Pfizer Produccion de vector de virus adeno-asociado en celulas de insecto.
US20240335564A1 (en) * 2021-11-02 2024-10-10 Gritgen Therapeutics Limited Isolated nucleic acid molecule and use thereof
CA3242402A1 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025226943A1 (en) 2024-04-25 2025-10-30 Masonic Medical Research Laboratory Polynucleotide for hepatocyte expression of vestigial like protein 4 and method of use thereof
WO2025235425A1 (en) * 2024-05-06 2025-11-13 Research Institute At Nationwide Children's Hospital Improved proviral plasmids
WO2025250457A1 (en) 2024-05-28 2025-12-04 University Of Rochester Enhanced brain transduction by gene therapeutics

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115141836A (zh) * 2015-10-28 2022-10-04 桑格摩生物治疗股份有限公司 肝特异性构建体、因子viii表达盒及其使用方法

Family Cites Families (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU5588298A (en) 1996-12-02 1998-06-29 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6719969B1 (en) * 1999-08-09 2004-04-13 The Regents Of The University Of Michigan Treatment of liver disease and injury with CXC chemokines
CA2394850C (en) 1999-12-06 2012-02-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
KR20020086508A (ko) 2000-02-08 2002-11-18 상가모 바이오사이언스 인코포레이티드 약물 발견용 세포
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
EP1476547B1 (en) 2002-01-23 2006-12-06 The University of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
CA2392863A1 (en) 2002-07-08 2004-01-08 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw A high capacity recombinant adenoviral vector for treatment of hemophilia a
EP2806025B1 (en) 2002-09-05 2019-04-03 California Institute of Technology Use of zinc finger nucleases to stimulate gene targeting
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
PL1675956T3 (pl) * 2003-10-21 2011-06-30 Merck Serono Sa Minimalna sekwencja DNA działająca jako insulator chromatyny i jej zastosowanie do wyrażania białek
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
WO2005080581A2 (en) * 2004-02-17 2005-09-01 University Of Florida Research Foundation, Inc. Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression
KR20070060115A (ko) 2004-09-16 2007-06-12 상가모 바이오사이언스 인코포레이티드 단백질 생산을 위한 조성물 및 방법
MX2007015540A (es) * 2005-06-10 2008-03-07 Genentech Inc Mejora de la expresion de anticuerpos utilizando vectores que contienen elementos aislantes.
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
EP1985631A1 (en) * 2007-04-20 2008-10-29 LFB Biotechnologies Demannosylated recombinant factor VIII for the treatment of patients with hemophiila A
WO2008133938A2 (en) 2007-04-26 2008-11-06 Sangamo Biosciences, Inc. Targeted integration into the ppp1r12c locus
EP2281050B1 (en) 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Linear donor constructs for targeted integration
EP4154903A1 (en) 2008-04-22 2023-03-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
EP3156494B8 (en) 2008-12-04 2018-09-19 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
EP3594333B1 (en) 2010-02-08 2023-11-01 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US8771985B2 (en) 2010-04-26 2014-07-08 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using zinc-finger nucleases
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド 新規のｄｎａ結合タンパク質及びその使用
CA2993567C (en) 2010-07-21 2022-06-28 Sangamo Biosciences, Inc. Methods and compositions for modification of a t-cell receptor gene
EP2627665B1 (en) * 2010-10-12 2015-12-16 The Children's Hospital Of Philadelphia Methods and compositions for treating hemophilia B
EP3366312A1 (en) * 2011-06-23 2018-08-29 Alnylam Pharmaceuticals, Inc. Serpina 1 sirnas: compositions of matter and methods of treatment
AU2012312260B2 (en) 2011-09-21 2017-08-31 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
JP6188703B2 (ja) 2011-10-27 2017-08-30 サンガモ セラピューティクス， インコーポレイテッド Ｈｐｒｔ遺伝子座を修飾するための方法および組成物
AU2012340213B2 (en) 2011-11-16 2017-12-07 Sangamo Therapeutics, Inc. Modified DNA-binding proteins and uses thereof
DK2804623T3 (da) * 2012-01-12 2019-11-11 Bioverativ Therapeutics Inc Kimære faktor viii-polypeptider og anvendelser deraf
DK3444342T3 (da) 2012-07-11 2020-08-24 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme
SI2890780T1 (sl) 2012-08-29 2020-11-30 Sangamo Therapeutics, Inc. Postopki in sestavki za zdravljenje genetskega stanja
JP2015529466A (ja) * 2012-09-14 2015-10-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター
EP3513802B1 (en) * 2012-10-26 2023-11-29 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
EP2911687B1 (en) * 2012-10-26 2019-02-13 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
WO2014089212A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
BR112015025041A2 (pt) * 2013-03-30 2017-09-12 Usha Biotech Ltd métodos e conceitos para expressar proteínas biologicamente ativas em células de mamíferos
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
RS61039B1 (sr) * 2013-09-12 2020-12-31 Biomarin Pharm Inc Aav vektori koji sadrže gen koji kodira faktor viii
DK3080274T3 (da) * 2013-12-09 2020-08-31 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til genom-manipulation
CN115074366A (zh) * 2015-04-16 2022-09-20 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
EP3322297B1 (en) 2015-07-13 2024-12-04 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
RU2742352C2 (ru) 2015-09-24 2021-02-05 Байомарин Фармасьютикал Инк. Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции
EP3374387A1 (en) 2015-11-13 2018-09-19 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115141836A (zh) * 2015-10-28 2022-10-04 桑格摩生物治疗股份有限公司 肝特异性构建体、因子viii表达盒及其使用方法

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