KR20250022906A - 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법 - Google Patents

간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법 Download PDF

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KR20250022906A
KR20250022906A KR1020257003908A KR20257003908A KR20250022906A KR 20250022906 A KR20250022906 A KR 20250022906A KR 1020257003908 A KR1020257003908 A KR 1020257003908A KR 20257003908 A KR20257003908 A KR 20257003908A KR 20250022906 A KR20250022906 A KR 20250022906A
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sequence
transgene
hfviii
expression construct
protein
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Korean (ko)
Inventor
브리짓 이. 라일리
게리 케이. 이
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상가모 테라퓨틱스, 인코포레이티드
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KR1020257003908A 2015-10-28 2016-07-13 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법 Pending KR20250022906A (ko)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
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
PCT/US2016/042099 WO2017074526A1 (en) 2015-10-28 2016-07-13 Liver-specific constructs, factor viii expression cassettes and methods of use thereof
KR1020187014472A KR102766526B1 (ko) 2015-10-28 2016-07-13 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법

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KR1020187014472A Active KR102766526B1 (ko) 2015-10-28 2016-07-13 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법

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CN (2) CN115141836B (enExample)
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Families Citing this family (87)

* 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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 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
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
EP3368664A4 (en) * 2015-10-28 2019-08-28 Sangamo Therapeutics, Inc. LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD 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
US11584780B2 (en) 2016-07-26 2023-02-21 Biomarin Pharmaceutical Inc. Adeno-associated virus capsid proteins
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CA3035534A1 (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
EP4667565A2 (en) 2016-10-20 2025-12-24 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
IL266862B2 (en) 2016-12-01 2024-01-01 Sangamo Therapeutics Inc Tau modulators and methods and preparations for their administration
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11560566B2 (en) 2017-05-12 2023-01-24 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)
MY208862A (en) * 2017-08-01 2025-06-04 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
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
EP3697907A1 (en) * 2017-10-17 2020-08-26 CRISPR Therapeutics AG 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
EP3737762A1 (en) 2018-01-12 2020-11-18 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
BR112020015798A2 (pt) 2018-02-01 2021-03-09 Homology Medicines, Inc. Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas
RU2020129448A (ru) 2018-02-08 2022-03-09 Сангамо Терапьютикс, Инк. Сконструированные специфические для мишени нуклеазы
MA51869A (fr) * 2018-02-16 2020-12-23 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique par ciblage du fibrinogène-alpha
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
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
KR20210008491A (ko) * 2018-05-14 2021-01-22 바이오마린 파머수티컬 인크. 청소년 대상체에서 aav 벡터의 안정한 발현
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN112639110A (zh) * 2018-06-22 2021-04-09 阿斯克肋匹奥生物制药公司 用于基因递送以在细胞内持续存在的载体
MX2021001375A (es) * 2018-08-03 2021-04-19 Sangamo Therapeutics Inc Parametros clinicos mejorados por la expresion del factor viii.
WO2020041773A1 (en) * 2018-08-24 2020-02-27 Spark Therapeutics, Inc. Optimized promoter sequences, intron-free expression constructs and methods of use
US12329857B2 (en) 2018-09-21 2025-06-17 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
WO2020072451A1 (en) * 2018-10-01 2020-04-09 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
US12318427B2 (en) 2018-10-02 2025-06-03 Sangamo Therapeutics, Inc. Methods and compositions for modulation of tau proteins
KR20210091167A (ko) 2018-10-16 2021-07-21 블루알렐, 엘엘씨 유전자에서 dna의 표적화 삽입을 위한 방법
MX2021004277A (es) 2018-10-18 2021-09-08 Intellia Therapeutics Inc Composiciones y metodos para expresar el factor ix.
WO2020084162A1 (en) * 2018-10-26 2020-04-30 Vrije Universiteit Brussel Liver-specific nucleic acid regulatory elements and methods and use thereof
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
AU2020205717B2 (en) 2019-01-11 2025-09-11 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
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2019428629A1 (en) 2019-02-06 2021-01-28 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
MA54951A (fr) 2019-02-15 2021-12-22 Bayer Healthcare Llc Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée
MA55313A (fr) 2019-03-13 2022-01-19 Generation Bio Co Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
AU2020253532B2 (en) * 2019-04-04 2024-06-20 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized coagulation factor 12 locus
EP3956349A1 (en) 2019-04-17 2022-02-23 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
CA3165015A1 (en) * 2020-01-16 2021-07-22 Matthias Klugmann Adeno associated virus based gene therapy for phenylketonuria
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
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
AU2021308681A1 (en) 2020-07-16 2023-03-09 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
MX2023005218A (es) 2020-11-03 2023-05-16 Pfizer Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico.
KR102536321B1 (ko) 2020-12-09 2023-05-26 재단법인 아산사회복지재단 인체 유래 유전자 발현 유도 모듈에 기반한 간 특이 유전자 발현 시스템
IL303737A (en) 2020-12-18 2023-08-01 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
KR20240023638A (ko) 2021-06-22 2024-02-22 화이자 인코포레이티드 곤충 세포에서의 아데노-연관 바이러스 벡터의 생산
WO2023078220A1 (zh) * 2021-11-02 2023-05-11 苏州华毅乐健生物科技有限公司 分离的核酸分子及其应用
KR20240123832A (ko) 2021-12-16 2024-08-14 아퀴타스 테라퓨틱스 인크. 지질 나노입자 제형에 사용하기 위한 지질
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

Family Cites Families (57)

* 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
AU776576B2 (en) 1999-12-06 2004-09-16 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
WO2003087341A2 (en) 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
EP1504092B2 (en) 2002-03-21 2014-06-25 Sangamo BioSciences, Inc. 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
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP1675956B1 (en) * 2003-10-21 2010-10-13 Merck Serono SA Minimal dna sequence acting as a chromatin insulator and its use in protein expression
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
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
CA2611465A1 (en) * 2005-06-10 2007-02-22 Genentech, Inc. Improving antibody expression using vectors containing insulator elements
EP2027262B1 (en) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variant foki cleavage half-domains
JP5551432B2 (ja) 2006-05-25 2014-07-16 サンガモ バイオサイエンシーズ， インコーポレイテッド 遺伝子不活性化のための方法と組成物
EP1985631A1 (en) * 2007-04-20 2008-10-29 LFB Biotechnologies Demannosylated recombinant factor VIII for the treatment of patients with hemophiila A
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
AU2009238629C1 (en) 2008-04-14 2015-04-30 Sangamo Therapeutics, Inc. Linear donor constructs for targeted integration
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
US8703489B2 (en) 2008-08-22 2014-04-22 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
ES2627552T3 (es) 2008-12-04 2017-07-28 Sigma Aldrich Company Edición de genoma en ratas usando nucleasas con dedos de cinc
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
EP2534173B1 (en) 2010-02-08 2019-09-11 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
CN102939377B (zh) 2010-04-26 2016-06-08 桑格摩生物科学股份有限公司 使用锌指核酸酶对Rosa位点进行基因组编辑
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
AU2011281062B2 (en) 2010-07-21 2015-01-22 Board Of Regents, The University Of Texas System Methods and compositions for modification of a HLA locus
US9175280B2 (en) 2010-10-12 2015-11-03 Sangamo Biosciences, Inc. Methods and compositions for treating hemophilia B
US20140235693A1 (en) * 2011-06-23 2014-08-21 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
KR102061557B1 (ko) 2011-09-21 2020-01-03 상가모 테라퓨틱스, 인코포레이티드 이식 유전자 발현의 조절을 위한 방법 및 조성물
AU2012328682B2 (en) 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
CA2854819C (en) 2011-11-16 2022-07-19 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof
RS59670B1 (sr) 2012-01-12 2020-01-31 Bioverativ Therapeutics Inc Himerni polipeptidi faktora viii i njihove upotrebe
EP3816281A1 (en) 2012-07-11 2021-05-05 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
KR20220164090A (ko) 2012-08-29 2022-12-12 상가모 테라퓨틱스, 인코포레이티드 유전적 병태를 치료하기 위한 방법 및 조성물
WO2014043131A1 (en) * 2012-09-14 2014-03-20 The Regents Of The University Of California Lentiviral vector for stem cell gene therapy of sickle cell disease
DK2911687T3 (da) * 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
US10398787B2 (en) * 2012-10-26 2019-09-03 Vrije Universiteit Brussel Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
AU2013355327A1 (en) 2012-12-05 2015-06-11 Sangamo Therapeutics, Inc. Methods and compositions for regulation of metabolic disorders
AU2014246731A1 (en) * 2013-03-30 2015-11-19 Usha Biotech Limited Methods and constructs for expressing biologically active proteins in mammalian cells
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
SI3044231T1 (sl) * 2013-09-12 2020-12-31 Biomarin Pharmaceutical Inc. Vektorji AAV, ki vsebujejo gen, ki kodira faktor VIII
CN105940013B (zh) * 2013-12-09 2020-03-27 桑格摩生物科学股份有限公司 用于治疗血友病的方法和组合物
ES3009565T3 (en) * 2015-04-16 2025-03-27 Univ Emory Recombinant promoters and vectors for protein expression in liver and use thereof
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
RU2020126034A (ru) 2015-09-24 2020-09-02 Байомарин Фармасьютикал Инк. Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции
EP3368664A4 (en) * 2015-10-28 2019-08-28 Sangamo Therapeutics, Inc. LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF
AU2016353353B2 (en) 2015-11-13 2020-07-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A

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