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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JP2021513871A5 (OSRAM
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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)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
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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JP2021513871A JP2021513871A (ja) 2021-06-03
JP2021513871A5 JP2021513871A5 (OSRAM) 2022-03-02
JP7244547B2 true JP7244547B2 (ja) 2023-03-22

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US (2) US11306329B2 (OSRAM)
EP (1) EP3755795A4 (OSRAM)
JP (1) JP7244547B2 (OSRAM)
CN (1) CN112041437A (OSRAM)
WO (1) WO2019161365A1 (OSRAM)

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US20250101465A1 (en) * 2021-05-11 2025-03-27 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 (148)

* 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
AU7906691A (en) 1990-05-23 1991-12-10 United States of America, as represented by the Secretary, U.S. Department of Commerce, The 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
EP0755454B1 (en) 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
WO1996008560A1 (en) 1994-09-16 1996-03-21 The Uab Research Foundation Transduction of genes into human hematopoietic stem cells using recombinant adeno-associated viral vectors
EP0785803A4 (en) 1994-09-23 2000-08-02 Gen Hospital Corp Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell
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
US6521225B1 (en) 1996-09-06 2003-02-18 Chiron Corporation AAV vectors
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6281009B1 (en) 1996-09-11 2001-08-28 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
WO1998024479A1 (en) 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
EP0946723A1 (en) 1996-12-18 1999-10-06 Targeted Genetics Corporation Recombinase-activatable aav packaging cassettes for use in the production of aav vectors
CA2275892A1 (en) 1996-12-23 1998-07-02 Gene-Cell, Inc. 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
ATE420157T1 (de) 1997-04-24 2009-01-15 Univ Washington Zielgerichtete genveraenderung mit parvoviralen vektoren
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
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
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
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
DE60034478T2 (de) 1999-11-16 2008-01-10 Genzyme Corp., Cambridge Vektoren und transgene mit regulatorischen elementen zur genverabreichung in leber
AU2001255575B2 (en) 2000-04-28 2006-08-31 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
AU2002306539A1 (en) 2001-02-16 2002-09-04 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
EP1395293B1 (en) 2001-05-14 2009-07-22 Gbp Ip, Llc Lentiviral vectors encoding clotting factors for gene therapy
WO2002097056A2 (en) 2001-05-31 2002-12-05 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
JP2005526001A (ja) 2001-10-04 2005-09-02 アグイラルーコルドバ，カリオス，エスツアルド 遺伝子療法用キメラ型ウイルスベクター
CA2361462A1 (en) 2001-11-07 2003-05-08 Katherine A. High Induction of tolerance to a therapeutic polypeptide
SG10202108118RA (en) 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
CA2369985A1 (en) 2002-01-18 2003-07-18 Duke University Generation of recombinant adeno-associated viral vectors by a complete adenovirus-mediated approach
WO2003093460A1 (en) 2002-04-29 2003-11-13 The Trustees Of The University Of Pennsylvania Method for direct rescue and amplification of integrated viruses from cellular dna of tissues
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
US7220577B2 (en) 2002-08-28 2007-05-22 University Of Florida Research Foundation, Inc. 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
PL2357189T3 (pl) 2003-06-19 2017-08-31 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
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
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
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
DK1804839T3 (da) 2004-09-22 2012-04-10 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
TWI293307B (en) 2004-09-30 2008-02-11 Ind Tech Res Inst A liver-specific chimeric regulatory sequence and use thereof
EP1871887A2 (en) 2005-03-09 2008-01-02 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
EP3085389A1 (en) 2005-04-07 2016-10-26 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
EP1928504B1 (en) 2005-08-19 2013-04-24 University of Technology, Sydney Liver-directed gene therapy
AU2006304997B2 (en) 2005-10-20 2012-03-01 Uniqure Ip B.V. Improved AAV vectors produced in insect cells
EP1981548A4 (en) 2006-01-20 2010-03-24 Univ North Carolina REINFORCED 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
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford 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
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
HUE030719T2 (en) 2007-04-09 2017-05-29 Univ Florida Raav vector compositions having tyrosine-modified capsid proteins and methods for use
EP1985631A1 (en) * 2007-04-20 2008-10-29 LFB Biotechnologies Demannosylated recombinant factor VIII for the treatment of patients with hemophiila A
US10696983B2 (en) 2007-05-30 2020-06-30 The Trustees Of The University Of Pennsylvania 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
US20090215879A1 (en) 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
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
EP2315833B8 (en) 2008-05-20 2015-05-27 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
ES2724122T3 (es) 2009-04-30 2019-09-06 Univ Pennsylvania Composiciones para dirigir células de las vías respiratorias de conducción que comprenden construcciones de virus adenoasociado
US8476418B2 (en) 2009-05-28 2013-07-02 Deutsches Krebsforschungszentrum Modified AAV capsid polypeptides
KR101812813B1 (ko) 2009-06-16 2017-12-27 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
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
ES2680915T3 (es) 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
WO2011117258A2 (en) 2010-03-22 2011-09-29 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
KR20130040844A (ko) 2010-03-29 2013-04-24 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 약학적으로 유발된 전이유전자 제거 시스템
US9839696B2 (en) * 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
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
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
US9409953B2 (en) 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
US10392632B2 (en) 2011-02-14 2019-08-27 The Children's Hospital Of Philadelphia AAV8 vector with enhanced functional activity and methods of use thereof
SG10202007803QA (en) 2011-02-17 2020-09-29 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
US9402919B2 (en) 2011-03-04 2016-08-02 Intrexon Corporation Vectors conditionally expressing protein
CN107012171B (zh) 2011-04-22 2021-07-13 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
EP3147295B2 (en) 2011-08-24 2023-11-22 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
US8895264B2 (en) 2011-10-27 2014-11-25 Sangamo Biosciences, Inc. Methods and compositions for modification of the HPRT locus
CA2856137A1 (en) 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
WO2013096955A1 (en) 2011-12-23 2013-06-27 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
KR102057540B1 (ko) 2012-02-17 2019-12-19 더 칠드런스 호스피탈 오브 필라델피아 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법
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
CN104619832B (zh) 2012-07-06 2021-03-16 衣阿华大学研究基金会 经修饰的腺伴随病毒载体组合物
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
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
EP2940131B1 (en) 2012-12-25 2019-02-20 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
ES2741502T3 (es) 2013-02-08 2020-02-11 Univ Pennsylvania Cápside de AAV8 modificada para la transferencia génica para terapias de retina
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
JP6591956B2 (ja) 2013-03-15 2019-10-16 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 Ｍｐｓ１を治療するための組成物および方法
AU2014229051B2 (en) 2013-03-15 2020-07-16 The Children's Hospital Of Philadelphia Vectors comprising stuffer/filler polynucleotide sequences and methods of use
KR102298579B1 (ko) 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
AU2014287005C1 (en) 2013-07-12 2021-06-03 The Children's Hospital Of Philadelphia AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies
PT3024498T (pt) 2013-07-22 2020-03-06 Childrens Hospital Philadelphia Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
CN110713995B (zh) 2013-10-17 2023-08-01 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
MX392639B (es) 2014-03-21 2025-03-24 Univ Leland Stanford Junior Vectores virales recombinantes para la integración de transgenes
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
US10876134B2 (en) 2014-12-16 2020-12-29 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
JP6865167B2 (ja) 2014-12-17 2021-04-28 フンダシオン パラ ラ インベスティガシオン メディカ アプリカダ ウィルソン病の処置に使用するための核酸構築物及び遺伝子治療ベクター
ES2784264T3 (es) 2014-12-17 2020-09-23 Fundacion Para La Investig Medica Aplicada Construcciones de ácido nucleico y vectores de terapia génica para su uso en el tratamiento de la enfermedad de Wilson y otras afecciones
WO2016127057A1 (en) 2015-02-06 2016-08-11 The University Of North Carolina At Chapel Hill Optimized human clotting factor viii gene expression cassettes and their use
WO2016146757A1 (en) 2015-03-17 2016-09-22 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
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
EP3283126B1 (en) 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
CN115141836B (zh) 2015-10-28 2024-08-27 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
WO2017100551A1 (en) 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION
EP3562514B1 (en) 2016-12-30 2026-04-29 The Trustees Of The University Of Pennsylvania Gene therapy for treating wilson's disease
FI3568483T3 (fi) 2017-01-10 2025-11-21 Childrens Medical Res Institute Polynukleotideja ja vektoreita siirtogeenien ilmentämiseen
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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