JP7244547B2 - Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 - Google Patents

Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 Download PDF

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JP7244547B2
JP7244547B2 JP2020566527A JP2020566527A JP7244547B2 JP 7244547 B2 JP7244547 B2 JP 7244547B2 JP 2020566527 A JP2020566527 A JP 2020566527A JP 2020566527 A JP2020566527 A JP 2020566527A JP 7244547 B2 JP7244547 B2 JP 7244547B2
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nucleotide sequence
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サスワティ・チャタジー
ローラ・ジェーン・スミス
ジェフ・リン・エルズワース
ヒラード・ルビン
ジェーソン・ボーク・ライト
ジェームズ・アンソニー・マクスウィゲン
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City of Hope
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JP2020566527A 2018-02-19 2019-02-19 Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 Active JP7244547B2 (ja)

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US201862632300P 2018-02-19 2018-02-19
US62/632,300 2018-02-19
US201862632919P 2018-02-20 2018-02-20
US62/632,919 2018-02-20
US201862672385P 2018-05-16 2018-05-16
US62/672,385 2018-05-16
PCT/US2019/018502 WO2019161365A1 (en) 2018-02-19 2019-02-19 Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof

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JP2021513871A5 JP2021513871A5 (enExample) 2022-03-02
JP7244547B2 true JP7244547B2 (ja) 2023-03-22

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EP (1) EP3755795A4 (enExample)
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WO2022241041A2 (en) * 2021-05-11 2022-11-17 City Of Hope Adeno-associated virus compositions and methods of use thereof
WO2024229281A1 (en) * 2023-05-02 2024-11-07 City Of Hope Adeno-associated virus compositions and methods of use thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150306250A1 (en) 2012-12-07 2015-10-29 Haplomics, Inc. Factor viii mutation repair and tolerance induction
JP2017529854A (ja) 2014-09-24 2017-10-12 シティ・オブ・ホープＣｉｔｙ ｏｆ Ｈｏｐｅ 高効率ゲノム編集のためのアデノ随伴ウイルスベクターバリアント及びその方法

Family Cites Families (149)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
DE4339922C1 (de) 1993-09-03 1994-10-06 Max Planck Gesellschaft Vektor für Leber-Gentherapie
US6268212B1 (en) 1993-10-18 2001-07-31 Amgen Inc. Tissue specific transgene expression
DE4407859C1 (de) 1994-03-04 1995-03-02 Max Planck Gesellschaft Vektor für die leberspezifische Gentherapie
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
AU3718595A (en) 1994-09-16 1996-03-29 Uab Research Foundation, The Transduction of genes into human hematopoietic stem cells using recombinant adeno-associated viral vectors
JPH10506530A (ja) 1994-09-23 1998-06-30 ザ ジェネラル ホスピタル コーポレーション 哺乳類細胞において外来遺伝子を発現する非哺乳類ｄｎａウイルスの使用
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US5650309A (en) 1995-05-16 1997-07-22 The Regents Of The University Of California Viral vectors
US7001764B2 (en) 1995-06-27 2006-02-21 Cell Genesys, Inc. Compositions comprising tissue specific adenoviral vectors
DE19525900C1 (de) 1995-07-15 1996-12-12 Max Planck Gesellschaft Leberspezifischer Adenovirus-Expressionsvektor
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5780447A (en) 1996-06-14 1998-07-14 St. Jude Children's Research Hospital Recombinant adeno-associated viral vectors
WO1998009524A1 (en) 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
WO1998012311A2 (en) 1996-09-11 1998-03-26 The General Hospital Corporation Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell
DE19644500C2 (de) 1996-10-25 1998-11-05 Deutsches Krebsforsch AAV-DNA mit Helfervirus-Sequenzen
FR2755699B1 (fr) 1996-11-08 1998-12-18 Rhone Poulenc Rorer Sa Nouvelles constructions et vecteurs pour l'expression ciblee et inductible des genes
FR2756297B1 (fr) 1996-11-22 1999-01-08 Centre Nat Rech Scient Procede de production de virus recombinants
AU5588298A (en) 1996-12-02 1998-06-29 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
JP2001506132A (ja) 1996-12-18 2001-05-15 ターゲティッド ジェネティクス コーポレイション Ａａｖベクターの産生における使用のためのリコンビナーゼ活性化可能ａａｖパッケージングカセット
EP0946718A1 (en) 1996-12-23 1999-10-06 Gene-Cell Nucleic acid constructs and uses thereof for direct nucleic acid incorporation into cells
US6153436A (en) 1997-01-10 2000-11-28 The Board Of Trustees Of The University Of Arkansas Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions
JP2001522244A (ja) 1997-04-24 2001-11-13 ユニバーシティ オブ ワシントン パルボウイルスベクターによる標的化した遺伝子改変
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
GB9715064D0 (en) 1997-07-17 1997-09-24 Ppl Therapeutics Scotland Ltd Protein expression
WO1999018227A1 (en) 1997-10-08 1999-04-15 Advanced Research And Technology Institute Chimeric parvovirus-based recombinant vector system that specifically targets the erythroid lineage
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
DE69918090T2 (de) 1998-04-24 2005-06-16 University Of Florida, Gainesville Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
WO1999064569A1 (en) 1998-06-10 1999-12-16 Advanced Research And Technology Institute Methods and compositions for generating recombinant adeno-associated virus vectors
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US6919209B1 (en) 1998-12-04 2005-07-19 City Of Hope Method of genetically modifying very primitive quiescent human hematopoietic stem cells
JP4693244B2 (ja) 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
US6610906B1 (en) 1999-06-09 2003-08-26 The Regents Of The University Of Michigan Nucleotide sequences for gene regulation and methods of use thereof
ES2284545T3 (es) 1999-11-16 2007-11-16 Genzyme Corporation Vectores y transgenes con elementos reguladores para la administracion de genes del higado.
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
US7351813B2 (en) 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
WO2002066611A2 (en) 2001-02-16 2002-08-29 The Board Of Trustees Of The Leland Stanford Junior University Minimal plasmid vectors that provide for persistent and high level gene expression and methods for using the same
DE10120265A1 (de) 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren
ATE437221T1 (de) 2001-05-14 2009-08-15 Gbp Ip Llc Lentivirale vektoren kodierend für gerinnungsfaktoren für die gentherapie
US8067156B2 (en) 2001-05-31 2011-11-29 The Rockefeller University Method for generating replication defective viral vectors that are helper free
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
US20030129203A1 (en) 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
EP1442125A4 (en) 2001-10-04 2004-12-08 Carlos Estuard Aguilar-Cordova CHIMERAL VIRUS VECTORS FOR GENE THERAPY
CA2361462A1 (en) 2001-11-07 2003-05-08 Katherine A. High Induction of tolerance to a therapeutic polypeptide
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
DK2573170T3 (en) 2001-12-17 2018-04-09 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use
CA2369985A1 (en) 2002-01-18 2003-07-18 Duke University Generation of recombinant adeno-associated viral vectors by a complete adenovirus-mediated approach
ATE348153T1 (de) 2002-04-29 2007-01-15 Univ Pennsylvania Methode für die direkte gewinnung und amplifikation von integrierten viren aus zellulärer gewebe-dna
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
AU2003265855A1 (en) 2002-08-28 2004-03-19 University Of Florida Modified aav
US20070202587A1 (en) 2002-09-23 2007-08-30 Applied Genetic Technologies Corporation Recombinant AAV production in mammalian cells
US7091029B2 (en) 2002-09-23 2006-08-15 Applied Genetics Technologies Corporation High titer recombinant AAV production
SI1633772T1 (sl) 2003-06-19 2016-06-30 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
US7482156B2 (en) 2003-10-15 2009-01-27 Cell Genesys, Inc. Hepatocellular carcinoma specific promoter and uses thereof
JP2007531537A (ja) 2004-04-06 2007-11-08 セダーズ−シナイ　メディカル　センター アポリポタンパク質ａ−ｉおよびアポリポタンパク質ａ−ｉｍｉｌａｎｏをコードする組換えアデノ随伴ウイルスベクターによる血管疾患の予防および処置
US8716461B2 (en) 2004-05-24 2014-05-06 Blood Systems, Inc. Human parvovirus
WO2006036502A2 (en) 2004-09-22 2006-04-06 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
TWI293307B (en) 2004-09-30 2008-02-11 Ind Tech Res Inst A liver-specific chimeric regulatory sequence and use thereof
WO2006096815A2 (en) 2005-03-09 2006-09-14 Board Of Regents, The University Of Texas System NOVEL hTMC PROMOTER AND VECTORS FOR THE TUMOR-SELECTIVE AND HIGH-EFFICIENT EXPRESSION OF CANCER THERAPEUTIC GENES
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
WO2007019646A1 (en) 2005-08-19 2007-02-22 University Of Technology, Sydney Liver-directed gene therapy
EP2311966A3 (en) 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) B.V. Improved AAV vectors produced in insect cells
WO2007084773A2 (en) 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2007127428A2 (en) 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
EP1985631A1 (en) * 2007-04-20 2008-10-29 LFB Biotechnologies Demannosylated recombinant factor VIII for the treatment of patients with hemophiila A
WO2008150459A1 (en) 2007-05-30 2008-12-11 The Trustees Of The University Of Pennsylvania A method for transducing cells with primary cilia
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009108274A2 (en) 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
WO2010011404A2 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP2425000B1 (en) 2009-04-30 2019-02-20 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
EP2435575A2 (en) 2009-05-28 2012-04-04 Deutsches Krebsforschungszentrum Modified aav capsid polypeptides
KR20180000341A (ko) 2009-06-16 2018-01-02 젠자임 코포레이션 재조합 ａａｖ 벡터에 대한 개선된 정제 방법
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
US20120244127A1 (en) 2009-10-01 2012-09-27 The Trustees Of The University Of Pennsylvania AAV Vectors Expressing SEC10 for Treating Kidney Damage
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
WO2011094198A1 (en) 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
US9546112B2 (en) 2010-03-22 2017-01-17 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US9839696B2 (en) * 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
US20120014932A1 (en) 2010-07-14 2012-01-19 Golden William J Methods for treating disorders of amino acid metabolism
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
EP2675484B1 (en) 2011-02-14 2018-05-30 The Children's Hospital of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
JP6189754B2 (ja) 2011-03-04 2017-08-30 イントレキソン コーポレーション タンパク質を条件的に発現するベクター
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
CA3099582A1 (en) 2011-10-27 2013-05-02 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
HK1202434A1 (en) 2011-11-22 2015-10-02 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US20140359799A1 (en) 2011-12-23 2014-12-04 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
MX367100B (es) 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
NZ701693A (en) 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
RU2679843C2 (ru) 2012-07-06 2019-02-13 Юниверсити Оф Айова Рисерч Фаундейшн Векторные композиции с модифицированным аденоассоциированным вирусом
EP2900686B1 (en) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
AU2013336601B2 (en) 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
US9255250B2 (en) 2012-12-05 2016-02-09 Sangamo Bioscience, Inc. Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene
US9938541B2 (en) 2012-12-25 2018-04-10 Takara Bio Inc. AAV variant
EP2943203A4 (en) 2013-01-11 2016-08-24 Scripps Research Inst METHOD AND COMPOSITIONS FOR INCREASING THE TRANSDUCTION EFFICIENCY OF RETROVIRAL VECTORS
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
HK1220488A1 (zh) 2013-03-15 2017-05-05 The Children's Hospital Of Philadelphia 含有填充者/填充物多核苷酸序列的载体及其使用方法
AU2014235096B2 (en) 2013-03-15 2020-03-19 The Trustees Of The University Of Pennsylvania Compositions and methods for treating MPSI
EP3492597A3 (en) 2013-05-21 2019-08-28 University of Florida Research Foundation, Inc. Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
HK1222673A1 (zh) 2013-07-12 2017-07-07 The Children's Hospital Of Philadelphia Aav载体和用於抗aav(腺相关病毒)中和抗体的检测
IL297919A (en) 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
EP3441468B1 (en) 2013-10-17 2021-05-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
BR112016019940A2 (pt) 2014-03-21 2017-10-24 Univ Leland Stanford Junior edição de genoma sem nucleases
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
WO2016100575A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
US11118238B2 (en) 2014-12-17 2021-09-14 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson's disease and other conditions
US11147887B2 (en) 2014-12-17 2021-10-19 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson disease
MX388739B (es) 2015-02-06 2025-03-20 Univ North Carolina Chapel Hill Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso.
CN104659074B (zh) 2015-03-17 2019-04-09 京东方科技集团股份有限公司 一种oled基板、其制造方法、面板及显示装置
US11007280B2 (en) 2015-03-17 2021-05-18 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
EP3283126B1 (en) 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
MY189674A (en) 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
WO2017100551A1 (en) 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION
BR112019013245A2 (pt) 2016-12-30 2020-02-11 The Trustees Of The University Of Pennsylvania Terapia gênica para tratamento da doença de wilson
PT3568483T (pt) 2017-01-10 2025-11-18 Childrens Medical Res Institute Polinucleótidos e vetores para a expressão de transgenes
WO2019010091A1 (en) 2017-07-06 2019-01-10 The Board Of Trustees Of The Leland Stanford Junior University METHODS AND COMPOSITIONS FOR FACILITATING HOMOLOGOUS RECOMBINATION

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150306250A1 (en) 2012-12-07 2015-10-29 Haplomics, Inc. Factor viii mutation repair and tolerance induction
JP2017529854A (ja) 2014-09-24 2017-10-12 シティ・オブ・ホープＣｉｔｙ ｏｆ Ｈｏｐｅ 高効率ゲノム編集のためのアデノ随伴ウイルスベクターバリアント及びその方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Homo sapiens coagulation factor VIII (F8), RefSeqGene on chromosome X NCBI Reference Sequence: NG_011403.1，2018年01月19日，2022年4月15日検索 <https://www.ncbi.nlm.nih.gov/nuccore/224586932?sat=46&satkey=166719631>
Scientific Reports，2016年，Vol.6, 18865, <DOI: 10.1038/srep18865>

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