ATE402254T1 - Aav5 vektoren und deren verwendung - Google Patents

Aav5 vektoren und deren verwendung

Info

Publication number
ATE402254T1
ATE402254T1 AT99926036T AT99926036T ATE402254T1 AT E402254 T1 ATE402254 T1 AT E402254T1 AT 99926036 T AT99926036 T AT 99926036T AT 99926036 T AT99926036 T AT 99926036T AT E402254 T1 ATE402254 T1 AT E402254T1
Authority
AT
Austria
Prior art keywords
aav5
vectors
aav5 vectors
present
particles
Prior art date
Application number
AT99926036T
Other languages
English (en)
Inventor
John Chiorini
Robert Kotin
Original Assignee
Us Gov Health & Human Serv
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Us Gov Health & Human Serv filed Critical Us Gov Health & Human Serv
Application granted granted Critical
Publication of ATE402254T1 publication Critical patent/ATE402254T1/de

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
AT99926036T 1998-05-28 1999-05-28 Aav5 vektoren und deren verwendung ATE402254T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US8702998P 1998-05-28 1998-05-28

Publications (1)

Publication Number Publication Date
ATE402254T1 true ATE402254T1 (de) 2008-08-15

Family

ID=22202669

Family Applications (1)

Application Number Title Priority Date Filing Date
AT99926036T ATE402254T1 (de) 1998-05-28 1999-05-28 Aav5 vektoren und deren verwendung

Country Status (9)

Country Link
US (1) US7479554B2 (de)
EP (1) EP1082413B1 (de)
JP (2) JP4060531B2 (de)
AT (1) ATE402254T1 (de)
AU (1) AU762220B2 (de)
CA (2) CA2745131C (de)
DE (1) DE69939169D1 (de)
ES (1) ES2313784T3 (de)
WO (1) WO1999061601A2 (de)

Families Citing this family (200)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
JP2002538770A (ja) * 1998-11-10 2002-11-19 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル ウイルスベクターとその製造及び投与の方法
AU7739000A (en) 1999-10-01 2001-05-10 University Of Florida Temperature-sensitive regulation of viral vector production
US6506600B2 (en) 2000-03-22 2003-01-14 University Of Arkansas Secreting products from skin by adeno-associated virus (AAV) gene transfer
US6855314B1 (en) * 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
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
EP1381276A4 (de) * 2001-04-13 2005-02-02 Univ Pennsylvania Verfahren zur behandlung oder zur entwicklungsverlangsamung von blindheit
DE10120265A1 (de) * 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren
US7271150B2 (en) 2001-05-14 2007-09-18 United States Of America, Represented By The Secretary, Department Of Health And Human Services Modified growth hormone
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
CN105671005B (zh) 2001-11-13 2020-09-15 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
AU2002360291A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
EP3339430A1 (de) 2001-12-17 2018-06-27 The Trustees of The University of Pennsylvania Adenoassoziierte virus(aav)-serotyp 9-sequenzen, vektoren damit und verwendungen davon
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
NZ545656A (en) 2003-09-01 2009-02-28 Amc Amsterdam AAV vectors for in vivo gene therapy of rheumatoid arthritis
EP2322638A1 (de) 2003-09-01 2011-05-18 Academisch Medisch Centrum AAV Vektoren für die in vivo Gentherapie von rheumatischer Arthritis
DK2292779T3 (en) 2003-09-30 2017-02-27 Univ Pennsylvania ADENOASS-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME AND APPLICATIONS THEREOF
WO2005056807A2 (en) * 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
WO2005058937A2 (en) 2003-12-12 2005-06-30 Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services A human cytotoxic t-lymphocyte epitope and its agonist epitope from the non-variable number of tandem repeat sequence of muc-1
WO2006029196A1 (en) * 2004-09-08 2006-03-16 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of H Ealth Transcytosis of adeno-associated viruses
WO2006066066A2 (en) 2004-12-15 2006-06-22 University Of North Carolina At Chapel Hill Chimeric vectors
EP4234687A2 (de) 2005-04-07 2023-08-30 The Trustees of the University of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
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
US8921050B2 (en) 2006-03-17 2014-12-30 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Methods of diagnosing renal cell carcinoma
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
ES2634118T3 (es) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
MX2011010097A (es) 2009-03-27 2011-10-19 Proyecto Biomedicina Cima Sl Metodos y composiciones para el tratamiento de cirrosis y fibrosis hepatica.
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
JP2014503173A (ja) 2009-11-05 2014-02-13 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 制御された発現系
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
EA202091105A1 (ru) * 2010-04-23 2020-12-30 Юниверсити Оф Флорида Рисерч Фаундейшн, Инк. Композиции гуанилатциклазы и способы лечения врожденного амавроза лебера типа 1 (lca1)
ES2698203T3 (es) 2010-04-23 2019-02-01 Univ Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
ES2661680T3 (es) 2011-04-21 2018-04-03 University Of Massachusetts Composiciones basadas en VAAr y métodos para tratar deficiencias de alfa-1 anti-tripsina
EP3693025B1 (de) 2011-04-22 2021-10-13 The Regents of The University of California Adenoassoziierte virus-virionen mit veränderlichem kapsid und verwendungsverfahren dafür
CA2864100A1 (en) * 2012-02-14 2013-08-22 The Regents Of The University Of California Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
EP2692868A1 (de) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adenoassoziierte virale (AAV) Vektoren für die Transduktion von Fettgewebe
CN104797593B (zh) 2012-09-28 2020-05-08 北卡罗来纳-查佩尔山大学 靶向少突胶质细胞的aav载体
WO2014059173A2 (en) 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
EP3517612A1 (de) 2013-03-15 2019-07-31 The University of North Carolina At Chapel Hill Synthetische adenoassoziierte virusinvertierte endwiederholungssequenzen
US20160122727A1 (en) 2013-06-13 2016-05-05 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
MX2016000345A (es) * 2013-07-12 2016-06-21 Philadelphia Children Hospital Vector aav y ensayo para anticuerpos neutralizantes anti-aav (virus adenoasociado).
US20160354489A1 (en) 2013-09-26 2016-12-08 Universitat Autònome de Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
CA2938669A1 (en) 2014-02-04 2015-08-13 Igenomx International Genomics Corporation Genome fractioning
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
DK3116900T3 (da) 2014-03-09 2020-09-28 Univ Pennsylvania Sammensætninger som kan anvendes til behandling af ornithintranscarbamylase (otc) defekt
EP3119797B1 (de) 2014-03-18 2020-12-23 University of Massachusetts Zusammensetzungen und verfahren zur behandlung von amyotropher lateralsklerose
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US9458464B2 (en) 2014-06-23 2016-10-04 The Johns Hopkins University Treatment of neuropathic pain
EP3194430A1 (de) 2014-09-16 2017-07-26 Universitat Autònoma De Barcelona Adeno-assoziierte virusvektoren zur gentheraüie von stoffwechselkrankheiten
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3795580A1 (de) 2014-10-03 2021-03-24 University of Massachusetts Hocheffiziente bibliotheksidentifizierte aav-vektoren
CA2964272A1 (en) * 2014-10-21 2016-04-28 Guangping Gao Recombinant aav variants and uses thereof
SG10202007103TA (en) 2014-11-05 2020-09-29 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA2975583A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
CN107532173A (zh) 2014-11-21 2018-01-02 北卡罗来纳-查佩尔山大学 靶向中枢神经系统的aav载体
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
EP3242945B1 (de) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Einzelvektor-genkonstrukt mit insulin- und glucokinasegenen
JP6938377B2 (ja) 2015-01-14 2021-09-22 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 標的化遺伝子移入のための方法および組成物
CN107531774B (zh) 2015-02-06 2021-12-14 北卡罗来纳大学查珀尔希尔分校 优化的人类凝血因子viii基因表达盒及其用途
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
US10968536B2 (en) 2015-02-25 2021-04-06 Jumpcode Genomics, Inc. Methods and compositions for sequencing
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
US20190000943A1 (en) * 2015-08-13 2019-01-03 Aavet Therapeutics, Llc Aav6 vectors for immunotherapy
EP3356390B1 (de) 2015-09-28 2021-01-20 The University of North Carolina at Chapel Hill Verfahren und zusammensetzungen für antikörperausweichende virusvektoren
US20190038659A1 (en) 2015-09-29 2019-02-07 The Usa, As Represented By The Secretary, Department Of Health And Human Services Methods of treating and preventing amyotrophic lateral sclerosis
US11253576B2 (en) 2015-10-22 2022-02-22 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
EP3364996B1 (de) 2015-10-22 2021-08-25 University of Massachusetts Gegen die prostata gerichtete adeno-assoziierte virusserotypvektoren
WO2017077451A1 (en) 2015-11-05 2017-05-11 Bamboo Therapeutics, Inc. Modified friedreich ataxia genes and vectors for gene therapy
EP3384035A4 (de) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. Assays zum nachweis von aav-neutralisierenden antikörpern
CA3012409A1 (en) * 2016-01-29 2017-08-03 Sirion Biotech Gmbh Aav-based conditional expression system
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
EP3411484B1 (de) 2016-02-05 2023-10-04 Emory University Injektion von einzelsträngigem oder selbstkomplementärem adeno-assoziiertem virus 9 in den liquor
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
EP4094780A3 (de) 2016-02-12 2023-02-08 University of Massachusetts Anti-angiogene mirna-therapeutika zur hemmung der neovaskularisation der hornhaut
US11339427B2 (en) 2016-02-12 2022-05-24 Jumpcode Genomics, Inc. Method for target specific RNA transcription of DNA sequences
WO2017147123A1 (en) 2016-02-22 2017-08-31 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i-associated blindness
MA43735A (fr) 2016-03-07 2018-11-28 Univ Iowa Res Found Expression médiée par aav utilisant un promoteur et un activateur synthétiques
EP3440210A4 (de) 2016-04-05 2019-11-27 University of Massachusetts Zusammensetzungen und verfahren zur selektiven hemmung der expression des grainyhead-like-proteins
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017190031A1 (en) 2016-04-28 2017-11-02 Indiana University Research And Technology Corporation Methods and compositions for resolving components of a virus preparation
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP4049683A1 (de) 2016-04-29 2022-08-31 Adverum Biotechnologies, Inc. Evasion von neutralisierenden antikörpern durch ein rekombinantes adeno-assoziiertes virus
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CN109476707B (zh) 2016-05-13 2022-12-02 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
CA3024448A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Modulatory polynucleotides
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
EP3469080A4 (de) 2016-06-13 2020-01-22 The University of North Carolina at Chapel Hill Optimierte cln1-gene und expressionskassetten sowie deren verwendung
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
US11866480B2 (en) 2016-07-26 2024-01-09 The University Of North Carolina At Chapel Hill Vector-mediated immune tolerance in the eye
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
US11578340B2 (en) 2016-10-13 2023-02-14 University Of Massachusetts AAV capsid designs
WO2018075798A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
WO2018083606A1 (en) 2016-11-01 2018-05-11 Novartis Ag Methods and compositions for enhancing gene editing
EP3570895A1 (de) 2017-01-17 2019-11-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Verfahren zur expression eines bestimmten polynukleotids in den zapfen-fotorezeptoren
EP3575398A4 (de) * 2017-01-30 2020-11-11 Nippon Medical School Foundation Mutant von capsid-protein des adeno-assoziierten virus (aav)
CA3054711A1 (en) 2017-03-15 2018-09-20 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
EP3388520A1 (de) 2017-04-11 2018-10-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Verfahren und pharmazeutische zusammensetzung zur reduzierung der expression von nkcc1 in einer darauf angewiesenen person
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
EP3618839A4 (de) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von amyotropher lateralsklerose (als)
CA3059213A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US20200207830A1 (en) 2017-07-27 2020-07-02 Novartis Ag Trem2 mutants resistant to sheddase cleavage
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
KR20200042935A (ko) * 2017-08-25 2020-04-24 오비드 테라퓨틱스 인크. 재조합 아데노-관련된 벡터들
WO2019060538A1 (en) 2017-09-20 2019-03-28 The Trustees Of Indiana University METHODS FOR LIPOPROTEIN RESOLUTION BY MASS SPECTROMETRY
FI3684423T3 (fi) 2017-09-20 2023-06-15 4D Molecular Therapeutics Inc Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä
US11739330B2 (en) 2017-09-22 2023-08-29 University Of Massachusetts SOD1 dual expression vectors and uses thereof
CN111479924A (zh) 2017-10-16 2020-07-31 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
EP3697908A1 (de) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Behandlung von amyotropher lateralsklerose (als)
EP3697448A1 (de) 2017-10-20 2020-08-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Verfahren zur expression eines bestimmten polynukleotids in den zapfen-fotorezeptoren einer person mit subretinaler abgabe einer therapeutisch wirksamen menge eines rekombinanten aav9-vektors
BR112020008835A2 (pt) 2017-11-07 2020-10-20 The University Of North Carolina At Chapel Hill genes de aga otimizados e cassetes de expressão e seu uso
EP4219695A3 (de) 2017-11-27 2024-01-17 4D Molecular Therapeutics Inc. Kapside mit adeno-assoziierten virenvarianten und verwendung zur hemmung der angiogenese
WO2019140233A1 (en) 2018-01-12 2019-07-18 The Trustees Of Indiana University Electrostatic linear ion trap design for charge detection mass spectrometry
JP2021511020A (ja) * 2018-01-17 2021-05-06 アドリーナス セラピューティクス, インコーポレイテッド 21−ヒドロキシラーゼ欠損症のためのアデノ随伴ウイルス遺伝子療法
WO2019165058A1 (en) 2018-02-21 2019-08-29 Alcyone Lifesciences, Inc. Fluid delivery systems and methods
EP3774852A1 (de) 2018-04-03 2021-02-17 Stridebio, Inc. Antikörpermeidende virale vektoren
EP3774854A1 (de) 2018-04-03 2021-02-17 Stridebio, Inc. Antikörperausweichende virusvektoren
US20210371469A1 (en) 2018-04-03 2021-12-02 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
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
UY38247A (es) 2018-05-30 2019-12-31 Novartis Ag Anticuerpos frente a entpd2, terapias de combinación y métodos de uso de los anticuerpos y las terapias de combinación
WO2019236143A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Apparatus and method for calibrating or resetting a charge detector
EP3803951A1 (de) 2018-06-04 2021-04-14 The Trustees of Indiana University Ladungsdetektionsmassenspektrometrie mit echtzeitanalyse und signaloptimierung
CN112673452A (zh) 2018-06-04 2021-04-16 印地安纳大学理事会 用于在静电线性离子阱中捕获离子的设备和方法
WO2019236139A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Interface for transporting ions from an atmospheric pressure environment to a low pressure environment
CA3102587A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Ion trap array for high throughput charge detection mass spectrometry
EP3807404A1 (de) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Manipulierte 5'-unübersetzte regionen (5' utr) zur herstellung von aav
CA3107462A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
US20210316012A1 (en) 2018-08-10 2021-10-14 The University Of North Carolina At Chapel Hill Optimized cln7 genes and expression cassettes and their use
CA3110345A1 (en) 2018-08-27 2020-03-05 Alcyone Lifesciences, Inc. Fluid delivery systems and methods
WO2020069029A1 (en) 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
EP3861113A1 (de) 2018-10-04 2021-08-11 Voyager Therapeutics, Inc. Verfahren zur messung des titers und der potenz von viralen vektorpartikeln
JP2022512621A (ja) 2018-10-05 2022-02-07 ボイジャー セラピューティクス インコーポレイテッド Aav産生タンパク質をコードする操作された核酸コンストラクト
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
JP2022505106A (ja) 2018-10-15 2022-01-14 ボイジャー セラピューティクス インコーポレイテッド バキュロウイルス/Sf9システムにおけるrAAVの大規模産生のための発現ベクター
CN113574632A (zh) 2018-11-20 2021-10-29 印地安纳大学理事会 用于单粒子质谱分析的轨道阱
AU2019392058A1 (en) 2018-12-03 2021-05-27 The Trustees Of Indiana University Apparatus and method for simultaneously analyzing multiple ions with an electrostatic linear ion trap
WO2020163379A1 (en) 2019-02-05 2020-08-13 Emendobio Inc. Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene
US20220154157A1 (en) 2019-02-06 2022-05-19 Emendobio Inc. New engineered high fidelity cas9
JP2022520875A (ja) 2019-02-25 2022-04-01 ノバルティス アーゲー Biettiクリスタリン網膜症を治療するための組成物及び方法
EP3931334A1 (de) 2019-02-25 2022-01-05 Novartis AG Zusammensetzungen und verfahren zur behandlung von bietti-kristall-dystrophie
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
WO2020205889A1 (en) * 2019-04-01 2020-10-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
JP2022530126A (ja) 2019-04-23 2022-06-27 アンセルム(アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル) 新規アデノ随伴ウイルス(aav)バリアント及び遺伝子治療のためのその使用
US11942317B2 (en) 2019-04-23 2024-03-26 The Trustees Of Indiana University Identification of sample subspecies based on particle mass and charge over a range of sample temperatures
WO2020219988A2 (en) 2019-04-26 2020-10-29 Sangamo Therapeutics, Inc. Engineering aav
WO2020232044A1 (en) * 2019-05-14 2020-11-19 Biomarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
SG11202112917PA (en) 2019-05-24 2021-12-30 Regeneron Pharma Modified viral particles and uses thereof
EP3990030A1 (de) 2019-06-27 2022-05-04 Pfizer Inc. Verfahren zur behandlung von duchenne-muskeldystrophie mittels aav-mini-dystrophin-gentherapie
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US10801042B1 (en) 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
JP2022548881A (ja) 2019-09-18 2022-11-22 ノバルティス アーゲー Entpd2抗体、組合せ療法並びに抗体及び組合せ療法を使用する方法
TW202124446A (zh) 2019-09-18 2021-07-01 瑞士商諾華公司 與entpd2抗體之組合療法
GB201913974D0 (en) 2019-09-27 2019-11-13 King S College London Vector
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease
JP2023515820A (ja) 2020-02-28 2023-04-14 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル メープルシロップ尿症の遺伝子治療
WO2021188960A1 (en) 2020-03-20 2021-09-23 University Of Florida Research Foundation, Inc. Gene therapy for cockayne syndrome
AU2021254272A1 (en) 2020-04-10 2022-10-27 Sola Biosciences Llc Compositions and methods for the treatment of protein aggregation disorders
BR112022021604A2 (pt) 2020-04-28 2022-12-06 Sola Biosciences Llc Composições e métodos para tratamento de proteinopatias associadas à tdp-43
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
EP4161953A1 (de) 2020-06-05 2023-04-12 SOLA Biosciences LLC Zusammensetzungen und verfahren zur behandlung von synukleinopathien
EP4240861A1 (de) 2020-11-03 2023-09-13 Pfizer Inc. Verfahren zur reinigung von aav-vektoren durch anionenaustauschchromatografie
US20240052322A1 (en) 2020-12-15 2024-02-15 Pfizer Inc. HILIC UPLC-MS Method For Separating and Analyzing Intact Adeno-Associated Virus Capsid Proteins
CA3205924A1 (en) 2020-12-23 2022-06-30 Pfizer Inc. Methods for purification of aav vectors by affinity chromatography
TW202242124A (zh) 2021-01-14 2022-11-01 美商史崔德生物公司 靶向t細胞之aav載體
WO2022208342A1 (en) 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
CN117377500A (zh) 2021-04-26 2024-01-09 阿雷克森国际制药有限公司 具有改善的组织向性的腺相关病毒载体衣壳
EP4349852A1 (de) 2021-05-28 2024-04-10 Shanghai Regenelead Therapies Co., Ltd. Rekombinantes adeno-assoziiertes virus mit veränderlichem kapsid und anwendung davon
US20220396798A1 (en) * 2021-06-12 2022-12-15 Shi-Lung Lin Novel mrna composition and production method for use in anti-viral and anti-cancer vaccines
WO2022269466A1 (en) 2021-06-22 2022-12-29 Pfizer Inc. Production of adeno-associated virus vector in insect cells
AR126407A1 (es) 2021-07-08 2023-10-11 Tenaya Therapeutics Inc Casetes de expresión optimizada para genoterapia
AU2022327583A1 (en) 2021-08-11 2024-02-22 King's College London Compositions and methods for improved treatment of disorders affecting the central nervous system
WO2023060221A2 (en) 2021-10-08 2023-04-13 Sola Biosciences Llc Compositions and methods for the treatment of proteopathies
CA3234720A1 (en) 2021-10-08 2023-04-13 Sola Biosciences Llc Compositions and methods for the treatment of p53-mediated cancers
US20230241118A1 (en) 2021-10-20 2023-08-03 University Of Rochester Rejuvenation treatment of age-related white matter loss
WO2023081633A1 (en) 2021-11-02 2023-05-11 University Of Rochester Tcf7l2 mediated remyelination in the brain
CA3233698A1 (en) 2021-11-04 2023-05-11 Regeneron Pharmaceuticals, Inc. Viral particles retargeted to skeletal muscle
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
TW202342759A (zh) 2022-02-04 2023-11-01 美商史崔德生物公司 重組腺相關病毒載體及其使用方法
WO2023201207A1 (en) 2022-04-11 2023-10-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2023220603A1 (en) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024026494A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Viral particles retargeted to transferrin receptor 1
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JPH09504173A (ja) * 1993-10-28 1997-04-28 ドイチェス クレプスフォルシュングスツェントルム シュティフトゥング デス エフェントリッヒェン レヒツ アデノ関連ウイルスとその早期流産の診断への利用
EP0755454B1 (de) * 1994-04-13 2008-02-13 The Rockefeller University Aav-vermittelte überbringung von dna in zellen des nervensystems
US6204059B1 (en) * 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
DE4436664A1 (de) * 1994-10-13 1996-07-04 Max Planck Gesellschaft Bereitstellung von rep-negativen AAV-Mutanten und hierfür verwendbare Zellen
US6342390B1 (en) * 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
US5843742A (en) 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
ATE258987T1 (de) 1995-08-03 2004-02-15 Avigen Inc Helfersystem zur wirkungssteigerung der aav- vektorproduktion
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus 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
WO1998041240A1 (en) * 1997-03-14 1998-09-24 The Children's Hospital Of Philadelphia Methods and compositions for use in gene therapy for treatment of hemophilia
IT1291135B1 (it) * 1997-04-08 1998-12-29 Angeletti P Ist Richerche Bio Vettori ricombinanti utilizzabili in terapia genica
ES2324540T3 (es) * 1998-05-27 2009-08-10 Genzyme Corporation Vectores aav para la fabricacion de medicamentos para la administracion potenciada por conveccion.
EP1080218A1 (de) * 1998-05-27 2001-03-07 University of Florida Methode zur darstellung von rekombinanten adeno-assoziierten virus zusammensetzungen mit hilfe von iodixanol gradienten
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
DE19849643A1 (de) 1998-10-29 2000-05-04 Deutsches Krebsforsch An das AAV-Kapsid bindender, den Zelltropismus verändernder Antikörper und Verfahren zum gerichteten Gentransfer
AU768729B2 (en) 1998-11-05 2004-01-08 Trustees Of The University Of Pennsylvania, The Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
AU4671300A (en) * 1999-04-30 2000-11-17 City Of Hope The use of adeno-associated virus (aav) deliver cytoprotective genes
US6468524B1 (en) * 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US20020076754A1 (en) * 2000-04-20 2002-06-20 Liangwu Sun Overcoming AAV vector size limitation through viral DNA hetero-dimerization
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
WO2002077221A1 (en) * 2001-03-27 2002-10-03 New York University Packaging cell lines for the continuous production of alphavirus vectors
CN105671005B (zh) 2001-11-13 2020-09-15 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
AU2002360291A1 (en) * 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
US20060127358A1 (en) 2002-05-01 2006-06-15 Nicholas Muzyczka Raav expression systems and methods for enhancing transduction of mammalian neural cells
US7419817B2 (en) * 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
ES2627913T3 (es) 2003-06-19 2017-08-01 Genzyme Corporation Viriones de AAV con inmunorreactividad reducida y usos de los mismos
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

Also Published As

Publication number Publication date
EP1082413A2 (de) 2001-03-14
DE69939169D1 (de) 2008-09-04
WO1999061601A9 (en) 2000-03-30
CA2745131A1 (en) 1999-12-02
ES2313784T3 (es) 2009-03-01
JP4060531B2 (ja) 2008-03-12
WO1999061601A3 (en) 2000-02-17
JP4191771B2 (ja) 2008-12-03
AU762220B2 (en) 2003-06-19
JP2007209347A (ja) 2007-08-23
WO1999061601A2 (en) 1999-12-02
CA2329060C (en) 2011-09-13
CA2745131C (en) 2016-08-09
US7479554B2 (en) 2009-01-20
AU4220599A (en) 1999-12-13
EP1082413B1 (de) 2008-07-23
JP2002516092A (ja) 2002-06-04
US20050255089A1 (en) 2005-11-17
CA2329060A1 (en) 1999-12-02

Similar Documents

Publication Publication Date Title
DE69939169D1 (de) Aav5 vektoren und deren verwendung
WO1998011244A3 (en) AAV4 vector and uses thereof
MX9603002A (es) Procedimiento para la preparacion de virus asociados a los adenovirus (aav), recombinantes, y usos de los mismos.
DK1127150T3 (da) Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse
ATE365808T1 (de) Transportproteine und deren verwendungen
UA42089C2 (uk) Протигрипозна вакцина на основі поверхневого антигену, спосіб її виробництва
WO2005056807A3 (en) Bovine adeno-associated viral (baav) vector and uses thereof
DE69435108D1 (de) Defekte adenovirus-vektoren und deren verwendung in der gentherapie
ATE437233T1 (de) Matrix-anheftungsregionen und verfahren zu deren verwendung
PT789766E (pt) Particulas quimericas do tipo papilomavirus
TR199901653T2 (xx) Fitalazinonlar.
DE69637256D1 (de) Synthese von Methoxynukleoside und enzymatische Nukleisäure Moleküle
PL332876A1 (en) Lentoviral vectors
BR9713338A (pt) Benzonaftiridinas como terapêuticos brÈnquicos.
DE69736840D1 (de) Synthese von nukleosiden und polynukleotiden
NO995182D0 (no) Pattedyrcytokinlignende faktor 7
ATE171212T1 (de) Corticotropin freisetzende rezeptoren des faktor 2
BR0010563A (pt) Composições isoladas de células da pele e métodos para seu uso
TR200002140T2 (tr) Naftil-ve anilid ile değiştirilmiş yeni sülfonamidler
DK1082423T3 (da) Cyclin E2-gener og -proteiner
MX9602557A (es) Proteinas de higado de mamifero y su uso en oncologia.
DE69809561T2 (de) Zusammensetzung und verfahren zur regulierung von zellproliferation und zelltod
ATE385517T1 (de) Interferon ähnliche moleküle, und deren verwendungen
DE69927795D1 (en) Temperaturstabile dna polymerase aus thermoanaerobacter thermohydrosulfuricus
DE69130845T2 (de) HLA-DR Antigen-Gen, dessen Nukleotidsequenz und Verwendung

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties