ATE315637T1 - Aav-capsidvehikel für den molekularen transport - Google Patents

Aav-capsidvehikel für den molekularen transport

Info

Publication number
ATE315637T1
ATE315637T1 AT95924751T AT95924751T ATE315637T1 AT E315637 T1 ATE315637 T1 AT E315637T1 AT 95924751 T AT95924751 T AT 95924751T AT 95924751 T AT95924751 T AT 95924751T AT E315637 T1 ATE315637 T1 AT E315637T1
Authority
AT
Austria
Prior art keywords
aav
production
capsids
aav capsid
vitro
Prior art date
Application number
AT95924751T
Other languages
English (en)
Inventor
Richard J Samulski
Forrest K Ferrari
Original Assignee
Univ Pittsburgh
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 Univ Pittsburgh filed Critical Univ Pittsburgh
Application granted granted Critical
Publication of ATE315637T1 publication Critical patent/ATE315637T1/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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10344Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • 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
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/14123Virus like particles [VLP]
    • 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
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10STECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S977/00Nanotechnology
    • Y10S977/70Nanostructure
    • Y10S977/788Of specified organic or carbon-based composition
    • Y10S977/802Virus-based particle
    • Y10S977/803Containing biological material in its interior
    • Y10S977/804Containing nucleic acid
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10STECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S977/00Nanotechnology
    • Y10S977/84Manufacture, treatment, or detection of nanostructure
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10STECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S977/00Nanotechnology
    • Y10S977/902Specified use of nanostructure
    • Y10S977/904Specified use of nanostructure for medical, immunological, body treatment, or diagnosis
    • Y10S977/918Immunological
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10STECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S977/00Nanotechnology
    • Y10S977/902Specified use of nanostructure
    • Y10S977/904Specified use of nanostructure for medical, immunological, body treatment, or diagnosis
    • Y10S977/92Detection of biochemical
AT95924751T 1994-06-30 1995-06-28 Aav-capsidvehikel für den molekularen transport ATE315637T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US08/268,430 US6204059B1 (en) 1994-06-30 1994-06-30 AAV capsid vehicles for molecular transfer

Publications (1)

Publication Number Publication Date
ATE315637T1 true ATE315637T1 (de) 2006-02-15

Family

ID=23022972

Family Applications (1)

Application Number Title Priority Date Filing Date
AT95924751T ATE315637T1 (de) 1994-06-30 1995-06-28 Aav-capsidvehikel für den molekularen transport

Country Status (8)

Country Link
US (3) US6204059B1 (de)
EP (1) EP0766569B1 (de)
JP (1) JPH10502526A (de)
AT (1) ATE315637T1 (de)
AU (1) AU705564B2 (de)
CA (1) CA2193802A1 (de)
DE (1) DE69534746T2 (de)
WO (1) WO1996000587A1 (de)

Families Citing this family (335)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
IL115880A (en) * 1995-11-06 2008-11-26 Hadasit Med Res Service Construct for infecting mammalian cells comprising the sv40 vp1 capsid protein and a histone - free constituent, methods of construction and uses thereof
PT914440E (pt) * 1996-04-19 2007-02-28 Univ Auburn Vacinação com áçido nucleico contra infecções parvovirais
US5866552A (en) * 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
AU771545B2 (en) * 1996-09-11 2004-03-25 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The AAV4 vector and uses thereof
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
JP2001506133A (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 組換えaavベクターの産生における使用のための、aavスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
DK1071806T3 (da) * 1998-04-24 2004-07-19 Univ Florida Rekombinant adeno-associeret virus-vektor, der koder for alpha-1-antitrypsin i genterapi
GB9811171D0 (en) * 1998-05-22 1998-07-22 Royal Free Hosp School Med Viral vector
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
JP4060531B2 (ja) * 1998-05-28 2008-03-12 アメリカ合衆国 Aav5ベクターおよびその使用
DE19827457C1 (de) 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
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
CA2348382C (en) * 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US20040043488A1 (en) * 1999-02-17 2004-03-04 University Of Pittsburgh Of The Commonwealth System Of Higher Education Adeno-associated viral gene-transfer vector system
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
US6498244B1 (en) * 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
DE19933719A1 (de) * 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
DE60131146T2 (de) * 2000-02-25 2008-03-06 Ludwig Institute For Cancer Research Material und verfahren die hybridvaskularendothelwachstumfaktoren dns und proteine enthalten und screeningverfahren für modulatoren
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
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
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
US6368602B1 (en) * 2000-06-16 2002-04-09 Hadasit Medical Research Services And Development Ltd Mucosal immunization against hepatitis A virus (HAV) through rectal administration of HAV vaccine
WO2002013758A2 (fr) * 2000-08-18 2002-02-21 Gencell S.A. Systeme de regulation in vivo de l'expression d'un transgene par inhibition conditionnelle
FR2813085A1 (fr) * 2000-08-18 2002-02-22 Aventis Pharma Sa Systeme de regulation in vivo de l'expression d'un transgene par inhibition conditionnelle
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
US7271002B2 (en) * 2001-11-09 2007-09-18 United States Of America, Represented By The Secretary, Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
JP4769417B2 (ja) 2001-12-17 2011-09-07 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 アデノ随伴ウイルス(aav)血清型9の配列、それを含むベクターおよびその使用
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
WO2003089612A2 (en) * 2002-04-17 2003-10-30 University Of Florida Research Foundation, Inc. IMPROVED rAAV VECTORS
ATE405295T1 (de) * 2002-05-01 2008-09-15 Univ Florida Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine
US20060093589A1 (en) * 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
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
WO2005017101A2 (en) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
AU2004272114B2 (en) * 2003-09-12 2010-11-04 Vertex Pharmaceuticals Incorporated Animal model for protease activity and liver damage
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
US7422741B2 (en) 2004-03-05 2008-09-09 Vegenics Limited VEGFR-3 fusion proteins
US7582442B2 (en) * 2004-03-16 2009-09-01 The Regents Of The University Of Michigan Methods and compositions for using aleveolar macrophage phospholipase A2
US7319015B2 (en) * 2004-03-16 2008-01-15 The Regents Of The University Of Michigan Methods and compositions for using alveolar macrophage phospholipase A2
US7964196B2 (en) * 2004-05-25 2011-06-21 Chimeros, Inc. Self-assembling nanoparticle drug delivery system
WO2006033689A2 (en) * 2004-06-18 2006-03-30 The University Of Montana Aav mediated gene delivery to cochlear cells
US20090233986A1 (en) * 2004-07-27 2009-09-17 Mount Sinai School Of Medicine Methods and compositions for using sax2
FI20050753A (fi) 2004-09-03 2006-03-04 Licentia Oy Uudet peptidit
CA2591544A1 (en) 2004-12-15 2006-06-22 The University Of North Carolina At Chapel Hill Chimeric vectors
WO2006101629A2 (en) * 2005-02-17 2006-09-28 Vertex Pharmaceuticals Incorporated SODIUM CHANNEL PROTEIN TYPE III α-SUBUNIT SPLICE VARIANT
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
US7972813B2 (en) * 2005-09-30 2011-07-05 Vertex Pharmaceuticals Incorporated Tetrodotoxin-resistant sodium channel alpha subunit
US20080200408A1 (en) * 2005-09-30 2008-08-21 Mccormack Kenneth Deletion mutants of tetrodotoxin-resistant sodium channel alpha subunit
CA2633468C (en) 2005-12-14 2014-02-18 Licentia Ltd Novel neurotrophic factor protein and uses thereof
WO2007084773A2 (en) * 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
US20090214496A1 (en) * 2006-01-30 2009-08-27 Licentia Ltd. Bmx/etk tyrosine kinase gene therapy materials and methods
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
CA2648581A1 (en) * 2006-04-07 2008-09-12 Chimeros, Inc. Compositions and methods for treating b- cell malignancies
EP2548578B1 (de) 2006-05-17 2014-08-20 The Ludwig Institute for Cancer Research Targeting der Regulierung von Fettsäuretransportern durch VEGF-B zur Modulation menschlicher Krankheiten
WO2007149852A2 (en) 2006-06-19 2007-12-27 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
WO2008024998A2 (en) 2006-08-24 2008-02-28 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
US20090226525A1 (en) * 2007-04-09 2009-09-10 Chimeros Inc. Self-assembling nanoparticle drug delivery system
EP2012122A1 (de) * 2007-07-06 2009-01-07 Medigene AG Strukturproteine von mutierten Parvoviren als Impfstoffe
FI20070808A0 (fi) 2007-10-25 2007-10-25 Mart Saarma GDNF:n silmukointivariantit ja niiden käytöt
US20090196854A1 (en) * 2008-02-04 2009-08-06 Kytos Biosystems S.A. Methods and compositions for use of crl 5803 cells for expression of biotherapeutics and encapsulated cell-based delivery
FI20080326A0 (fi) 2008-04-30 2008-04-30 Licentia Oy Neurotroofinen tekijä MANF ja sen käytöt
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8052970B2 (en) * 2008-06-30 2011-11-08 The Regents Of The University Of Michigan Lysosomal phospholipase A2 (LPLA2) activity as a diagnostic and therapeutic target for identifying and treating systemic lupus erythematosis
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
WO2010107778A1 (en) 2009-03-18 2010-09-23 Wake Forest University Health Sciences Flagellin fusion proteins and use thereof to induce immune responses against pseudomonas aeruginosa
WO2010120874A2 (en) 2009-04-14 2010-10-21 Chimeros, Inc. Chimeric therapeutics, compositions, and methods for using same
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US20120135037A1 (en) 2009-06-01 2012-05-31 Mizel Steven B Flagellin fusion proteins and conjugates comprising pneumococcus antigens and methods of using the same
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
CA2793633A1 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
WO2011133901A2 (en) 2010-04-23 2011-10-27 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
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
US9610354B2 (en) 2011-04-18 2017-04-04 National Center Of Neurology And Psychiatry Drug delivery particle and method for producing the same
EP3318635A1 (de) 2011-04-21 2018-05-09 University of Massachusetts Raav-basierte zusammensetzungen und verfahren zur behandlung von alpha-1-anti-trypsin-mangel
ES2748577T3 (es) 2011-05-19 2020-03-17 Univ Michigan Regents Agentes de fijación de la integrina alfa-2 y uso de los mismos para inhibir la proliferación de las células de cáncer
US8865188B2 (en) 2011-09-09 2014-10-21 Biomed Realty, L.P. Methods and compositions for controlling assembly of viral proteins
EP2785683B1 (de) 2011-11-30 2020-02-05 Ludwig Institute for Cancer Research Ltd. Inkt-zellmodulatoren und verwendungsverfahren dafür
GB201120860D0 (en) 2011-12-05 2012-01-18 Cambridge Entpr Ltd Cancer immunotherapy
JP5998229B2 (ja) 2011-12-08 2016-09-28 ビロベック・インコーポレイテッドVirovek,Inc. 毒性遺伝子を保有するベクター、そのための方法および使用
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
EP3818991A3 (de) 2012-06-19 2021-07-14 University of Florida Research Foundation, Inc. Zusammensetzungen und verfahren zur behandlung von krankheiten
EP2692868A1 (de) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adenoassoziierte virale (AAV) Vektoren für die Transduktion von Fettgewebe
PT2892617T (pt) 2012-09-06 2018-10-18 Univ Chicago Polinucleótidos antisenso para induzir o salto de exão e métodos de tratamentos de distrofias
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
CA2901226C (en) 2013-02-18 2020-11-17 Vegenics Pty Limited Vascular endothelial growth factor binding proteins
US10385112B2 (en) 2013-03-14 2019-08-20 Asklepios Biopharmaceutical, Inc. Modified soluble VEGF receptor-1 genes and vectors for gene therapy
EP2970966A4 (de) 2013-03-15 2016-10-26 Univ North Carolina Synthetische adenoassoziierte virusinvertierte endwiederholungssequenzen
CA2904396A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
WO2014191630A2 (en) 2013-05-28 2014-12-04 Helsingin Yliopisto Non-human animal model encoding a non-functional manf gene
PL3044231T3 (pl) 2013-09-12 2021-01-11 Biomarin Pharmaceutical Inc. Wektory aav zawierające gen kodujący czynnik viii
HUE052676T2 (hu) 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
SI3116900T1 (sl) 2014-03-09 2021-02-26 The Trustees Of The University Of Pennsylvania Sestavki uporabni pri zdravljenju pomanjkanja ornitin transkarbamilaze(OTC)
EP3750907A3 (de) 2014-03-18 2021-04-28 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
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
ES2876409T3 (es) 2014-04-25 2021-11-12 Univ Pennsylvania Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
MY186389A (en) 2014-05-13 2021-07-22 Univ Pennsylvania Compositions comprising aav expressing dual antibody constructs and uses thereof
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるaavベクター
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
AU2015346162B2 (en) 2014-11-14 2022-02-10 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
RU2727015C2 (ru) 2014-11-21 2020-07-17 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Векторы aav, нацеленные на центральную нервную систему
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
WO2016110518A1 (en) 2015-01-07 2016-07-14 Universitat Autònoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes
EP3245220B1 (de) 2015-01-14 2023-09-20 The University of North Carolina at Chapel Hill Verfahren und zusammensetzungen für gezielten gentransfer
US10308705B2 (en) 2015-02-06 2019-06-04 The University Of North Carolina At Chapel Hill Optimized human clotting factor VIII gene expression cassettes and their use
EP3256487A4 (de) 2015-02-09 2018-07-18 Duke University Zusammensetzungen und verfahren zur epigenombearbeitung
EP3256170B1 (de) 2015-02-13 2020-09-23 University of Massachusetts Zusammensetzungen und verfahren zur transitorischen freisetzung von polypeptiden
ES2846902T3 (es) 2015-04-08 2021-07-30 Univ Chicago Composiciones y procedimientos para corregir la distrofia muscular de cinturas tipo 2C mediante omisión de exones
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
JP6851319B2 (ja) 2015-04-27 2021-03-31 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 ヒト疾患のCRISPR/Cas9媒介性の修正のためのデュアルAAVベクター系
WO2016200543A2 (en) 2015-05-13 2016-12-15 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-inluenza antibodies and methods of use thereof
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
ES2929110T3 (es) 2015-08-25 2022-11-24 Univ Duke Composiciones y métodos para mejorar la especificidad en ingeniería genética usando endonucleasas guiadas por ARN
CA2995733A1 (en) 2015-08-31 2017-03-09 The Trustees Of The University Of Pennsylvania Aav-epo for treating companion animals
BR112018006074A2 (pt) 2015-09-24 2018-10-09 Biomarin Pharm Inc vetores de fator viii de vírus adeno-associado, partículas virais associadas e formulações terapêuticas compreendendo as mesmas
RU2727411C2 (ru) 2015-09-24 2020-07-21 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Композиция и способ для лечения заболевания, опосредованного комплементом
PL3356390T3 (pl) 2015-09-28 2021-07-05 The University Of North Carolina At Chapel Hill Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał
WO2017062750A1 (en) 2015-10-09 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating stargardt's disease and other ocular disorders
US11970710B2 (en) 2015-10-13 2024-04-30 Duke University Genome engineering with Type I CRISPR systems in eukaryotic cells
US10968253B2 (en) 2015-10-20 2021-04-06 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
DK3364997T3 (da) 2015-10-22 2024-04-22 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EP4316512A3 (de) 2015-10-28 2024-04-24 The Trustees of The University of Pennsylvania Intrathekale verabreichung von adeno-assoziierten viralen vektoren zur gentherapie
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
RU2021102893A (ru) 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
US11820972B2 (en) 2015-11-12 2023-11-21 Research Institute At Nationwide Children's Hospital Methods of treating muscular dystrophy
CA3008142A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
CA3007330A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Composition for treatment of crigler-najjar syndrome
WO2017106202A2 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
WO2017165859A1 (en) * 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
WO2017176929A1 (en) 2016-04-05 2017-10-12 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US11413356B2 (en) 2016-04-15 2022-08-16 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
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448987A4 (de) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
KR20230062878A (ko) 2016-07-08 2023-05-09 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
TN2019000047A1 (en) * 2016-08-15 2020-07-15 Genzyme Corp Methods for detecting aav
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
EP3548065B1 (de) 2016-12-01 2022-11-09 INSERM - Institut National de la Santé et de la Recherche Médicale Pharmazeutische zusammensetzungen zur behandlung von degenerativen netzhauterkrankungen
WO2018152485A1 (en) 2017-02-20 2018-08-23 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SG11201907611WA (en) 2017-02-28 2019-09-27 Univ Pennsylvania Influenza vaccines based on aav vectors
RS65241B1 (sr) 2017-02-28 2024-03-29 Univ Pennsylvania Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe
CA3054136A1 (en) 2017-03-01 2018-09-07 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
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
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
US11879133B2 (en) 2017-04-24 2024-01-23 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)治療組成物および方法
WO2018208972A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
JP7330899B2 (ja) 2017-05-10 2023-08-22 マサチューセッツ アイ アンド イヤー インファーマリー ウイルスのアセンブリ活性化タンパク質(aap)依存性を改変する方法および組成物
WO2018209113A1 (en) * 2017-05-10 2018-11-15 University Of Utah Research Foundation Compositions and methods of use of arc capsids
WO2018209205A1 (en) 2017-05-11 2018-11-15 The Trustees Of The University Of Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses
US20200102361A1 (en) 2017-05-24 2020-04-02 Universitat Autònoma De Barcelona Viral expression construct comprising a fibroblast growth factor 21 (fgf21) coding sequence
CA3098592A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
EP3638316A4 (de) 2017-06-14 2021-03-24 The Trustees Of The University Of Pennsylvania Gentherapie für augenerkrankungen
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
KR20200044793A (ko) 2017-08-03 2020-04-29 보이저 테라퓨틱스, 인크. Aav의 전달을 위한 조성물 및 방법
WO2019053272A1 (en) 2017-09-15 2019-03-21 King's College London COMPOSITIONS AND METHODS FOR IMPROVING T GAMMA DELTA LYMPHOCYTES IN INTESTINE
WO2019060538A1 (en) 2017-09-20 2019-03-28 The Trustees Of Indiana University METHODS FOR LIPOPROTEIN RESOLUTION BY MASS SPECTROMETRY
WO2019060686A1 (en) 2017-09-22 2019-03-28 University Of Massachusetts NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF
JP2021502060A (ja) 2017-10-16 2021-01-28 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)の治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
KR20200098481A (ko) 2017-10-16 2020-08-20 비게네론 게엠베하 Aav 벡터
CN113005123A (zh) * 2017-10-23 2021-06-22 普利维尔治疗公司 用于神经变性疾病的基因疗法
BR112020012336A2 (pt) 2017-12-19 2021-03-30 Akouos, Inc. Administração de anticorpos terapêuticos mediada por aav para o ouvido interno
EP3738137A1 (de) 2018-01-12 2020-11-18 The Trustees of Indiana University Konstruktion einer elektrostatischen linearen ionenfalle für massenspektrometrie mit ladungsdetektion
EP3762500A1 (de) 2018-03-06 2021-01-13 Voyager Therapeutics, Inc. Aus insektenzelle hergestellte teilweise selbstkomplementäre aav-genome
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
US20210371471A1 (en) 2018-04-03 2021-12-02 Stridebio, Inc. Antibody-evading virus vectors
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
IT201800004253A1 (it) 2018-04-05 2019-10-05 Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria.
AU2019257755A1 (en) 2018-04-27 2020-11-12 Voyager Therapeutics, Inc. Methods for measuring the potency of AADC viral vectors
SG11202010830WA (en) 2018-05-09 2020-11-27 Biomarin Pharm Inc Methods of treating phenylketonuria
US20200069819A1 (en) 2018-05-14 2020-03-05 Biomarin Pharmaceutical Inc. Stable expression of aav vectors in juvenile subjects
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20210214749A1 (en) 2018-05-16 2021-07-15 Voyager Therapeutics, Inc. Directed evolution
WO2019222441A1 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
WO2019236143A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Apparatus and method for calibrating or resetting a charge detector
JP7398811B2 (ja) 2018-06-04 2023-12-15 ザ・トラスティーズ・オブ・インディアナ・ユニバーシティー 高スループット電荷検出質量分光分析のためのイオン・トラップ・アレイ
US11227758B2 (en) 2018-06-04 2022-01-18 The Trustees Of Indiana University Apparatus and method for capturing ions in an electrostatic linear ion trap
CN112673451A (zh) 2018-06-04 2021-04-16 印地安纳大学理事会 具有实时分析和信号优化的电荷检测质谱分析
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
EP3807404A1 (de) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Manipulierte 5'-unübersetzte regionen (5' utr) zur herstellung von aav
CN112567035A (zh) 2018-07-02 2021-03-26 沃雅戈治疗公司 肌萎缩侧索硬化症及脊髓相关病症的治疗
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
KR20210068068A (ko) 2018-09-28 2021-06-08 보이저 테라퓨틱스, 인크. 조작된 프로모터를 갖는 프라탁신 발현 구축물 및 그의 사용 방법
CA3114175A1 (en) 2018-10-01 2020-04-09 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
CN113166208A (zh) 2018-10-02 2021-07-23 沃雅戈治疗公司 重新定向aav衣壳的嗜性
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
TW202028458A (zh) 2018-10-05 2020-08-01 美商航海家醫療公司 編碼腺相關病毒(aav)生產蛋白之經基因工程化核酸構築體
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3867389A1 (de) 2018-10-15 2021-08-25 Voyager Therapeutics, Inc. Expressionsvektoren zur massenproduktion von raav im baculovirus/sf9-system
AU2019384065A1 (en) 2018-11-20 2021-06-03 The Trustees Of Indiana University Orbitrap for single particle mass spectrometry
CN113227126A (zh) 2018-11-26 2021-08-06 巴塞罗那自治大学 成纤维细胞生长因子21(fgf21)基因治疗
CN113228226A (zh) 2018-12-03 2021-08-06 印地安纳大学理事会 利用静电线性离子阱同时分析多个离子的设备和方法
AU2020208467A1 (en) 2019-01-18 2021-08-05 Voyager Therapeutics, Inc. Methods and systems for producing AAV particles
US20220136008A1 (en) 2019-02-22 2022-05-05 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
US20220118108A1 (en) 2019-02-26 2022-04-21 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
WO2020219527A1 (en) 2019-04-23 2020-10-29 The Trustees Of Indiana University Identification of sample subspecies based on particle charge behavior under structural change-inducing sample conditions
AU2020263392A1 (en) 2019-04-26 2021-10-28 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV2.5 vector
TW202106879A (zh) 2019-04-29 2021-02-16 美商航海家醫療公司 於生物反應器中生產經桿狀病毒感染之昆蟲細胞(biic)之系統及方法
WO2020227515A1 (en) 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
US20200362368A1 (en) 2019-05-14 2020-11-19 Biomarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
CN113891726A (zh) 2019-05-31 2022-01-04 巴塞罗那自治大学 胰岛素基因疗法
KR20220031000A (ko) 2019-07-03 2022-03-11 각꼬호우징 닛뽄 이까다이가꾸 핵산 봉입aav 중공입자의 제조방법
EP4007814A1 (de) 2019-07-26 2022-06-08 Akouos, Inc. Verfahren zur behandlung von hörverlust unter verwendung eines sekretierten zielproteins
EP4010465A1 (de) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Zellkulturmedium zur verwendung bei der herstellung von gentherapieprodukten in bioreaktoren
US20220364114A1 (en) 2019-08-26 2022-11-17 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
JP2022549679A (ja) 2019-09-27 2022-11-28 バイオマリン ファーマシューティカル インコーポレイテッド サイズ排除クロマトグラフィー及び多角度光散乱技術を使用した遺伝子療法ウイルス粒子のキャラクタリゼーション
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
WO2021097157A1 (en) 2019-11-14 2021-05-20 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with liver-specific gene therapy vectors
CA3164189A1 (en) 2019-12-10 2021-06-17 Takeda Pharmaceutical Company Limited Adeno associated virus vectors for the treatment of hunter disease
JP2023512831A (ja) * 2020-02-07 2023-03-29 ヴェクプロバイオ インコーポレイテッド 植物において使用される組換えアデノ随伴ウイルスベクター
WO2021183895A1 (en) 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
EP4121168A1 (de) 2020-03-20 2023-01-25 University of Florida Research Foundation, Inc. Gentherapie für das schabyne-syndrom
US20230131352A1 (en) 2020-04-01 2023-04-27 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
AR122409A1 (es) 2020-04-03 2022-09-07 Biomarin Pharm Inc Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas
BR112022020753A2 (pt) 2020-04-15 2022-12-20 Voyager Therapeutics Inc Compostos de ligação a tau
GB202005732D0 (en) 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
WO2021230987A1 (en) 2020-05-13 2021-11-18 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
CA3183171A1 (en) 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating slc26a4-associated hearing loss
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
JP2023528590A (ja) 2020-05-26 2023-07-05 ウニベルシダッド アウトノマ デ バルセロナ 中枢神経系障害に対する線維芽細胞増殖因子21(fgf21)遺伝子治療
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
US20240018524A1 (en) 2020-07-10 2024-01-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and compositions for treating epilepsy
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
US20230227802A1 (en) 2020-07-27 2023-07-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
EP4192514A1 (de) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Zellkulturmedium zur verwendung bei der herstellung von gentherapieprodukten in bioreaktoren
CA3190399A1 (en) 2020-08-24 2022-03-03 James M. Wilson Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
CA3189107A1 (en) 2020-08-26 2022-03-03 James M. Wilson Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
IL301643A (en) 2020-10-07 2023-05-01 Regenxbio Inc Gene therapy for ocular manifestations of CLN2 disease
EP4228610A1 (de) 2020-10-13 2023-08-23 Kriya Therapeutics, Inc. Virale vektorkonstrukte zur abgabe von cytokin-codierenden nukleinsäuren und verwendungen davon zur behandlung von krebs
AU2021359874A1 (en) 2020-10-18 2023-05-25 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
JP2023547992A (ja) 2020-10-28 2023-11-15 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 二重グリカン結合aav2.5ベクターのための方法および組成物
WO2022093769A1 (en) 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
EP4236975A1 (de) 2020-10-29 2023-09-06 The Trustees of The University of Pennsylvania Aav-kapside und diese enthaltende zusammensetzungen
AU2021372262A1 (en) 2020-11-02 2023-06-01 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
AU2021375404A1 (en) 2020-11-03 2023-06-08 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
AR124216A1 (es) 2020-12-01 2023-03-01 Univ Pennsylvania Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen
PE20240115A1 (es) 2020-12-01 2024-01-22 Akouos Inc Construcciones de anticuerpos anti-vegf y metodos relacionados para el tratamiento de los sintomas asociados al schwannoma vestibular
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of AAV vectors by affinity chromatography
EP4271419A1 (de) 2020-12-29 2023-11-08 Akouos, Inc. Zusammensetzungen und verfahren zur behandlung von clrn1-assoziiertem hörverlust und/oder sehverlust
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
WO2022164923A1 (en) 2021-01-26 2022-08-04 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same
EP4284440A1 (de) 2021-01-30 2023-12-06 Universitat Autònoma De Barcelona Gentherapie für monogene diabetes
US20240091380A1 (en) 2021-02-01 2024-03-21 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
BR112023016983A2 (pt) 2021-02-26 2023-11-07 Takeda Pharmaceuticals Co Vetor de vírus adeno-associado recombinante, método para tratar doença de fabry, composição farmacêutica, célula, e, método de expressão da enzima ¿-gal em uma célula
US11760788B2 (en) 2021-03-02 2023-09-19 Pathways Neuro Pharma, Inc. Neuroreceptor compositions and methods of use
EP4301861A1 (de) 2021-03-03 2024-01-10 Shape Therapeutics Inc. Auxotrophe zellen zur virusproduktion sowie zusammensetzungen und verfahren zur herstellung
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187679A1 (en) 2021-03-04 2022-09-09 Kriya Therapeutics, Inc. Viral vector constructs incorporating dna for inhibiting toll like receptors and methods of using the same
EP4323520A1 (de) 2021-04-12 2024-02-21 The Trustees of The University of Pennsylvania Zusammensetzungen zur behandlung von spinaler und bulbusarer muskelatrophie (sbma)
WO2022221462A1 (en) 2021-04-13 2022-10-20 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic vlcad or mcad and methods of using the same
KR20240000580A (ko) 2021-04-23 2024-01-02 유니버시티 오브 로체스터 레트로바이러스 인테그라제-Cas 융합 단백질을 이용한 직접 비상동 DNA 삽입에 의한 게놈 편집 및 치료 방법
EP4089171A1 (de) 2021-05-12 2022-11-16 Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) Rekombinante tert-codierende virale genome und vektoren
EP4359547A1 (de) 2021-06-22 2024-05-01 Pfizer Inc. Herstellung eines adeno-assoziierten virusvektors in insektenzellen
WO2023017098A2 (en) 2021-08-11 2023-02-16 King's College London Compositions and methods for improved treatment of disorders affecting the central nervous system
WO2023023220A1 (en) 2021-08-20 2023-02-23 Alexion Pharmaceuticals, Inc. Methods for treating sickle cell disease or beta thalassemia using a complement alternative pathway inhibitor
WO2023023227A1 (en) 2021-08-20 2023-02-23 Alexion Pharmaceuticals, Inc. Methods for treating sickle cell disease or beta thalassemia using complement alternative pathway inhibitors
WO2023034996A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034989A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034990A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034994A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034997A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034980A1 (en) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023044483A2 (en) 2021-09-20 2023-03-23 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023056329A1 (en) 2021-09-30 2023-04-06 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
WO2023056436A2 (en) 2021-10-01 2023-04-06 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations
WO2023056399A1 (en) 2021-10-02 2023-04-06 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
TW202334436A (zh) 2021-11-02 2023-09-01 美商航海家醫療公司 Aav衣殼變異體及其用途
WO2023083750A1 (en) 2021-11-09 2023-05-19 The Francis Crick Institute Limited Viral adaptors and uses thereof
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023091948A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
WO2023111580A1 (en) 2021-12-16 2023-06-22 University Of Dundee Targeted degradation of alpha-synuclein
WO2023133561A1 (en) 2022-01-09 2023-07-13 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic anti-igf-1r antibodies and methods of using the same
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
WO2023150142A1 (en) 2022-02-02 2023-08-10 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
WO2023150687A1 (en) 2022-02-04 2023-08-10 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors, and methods of use thereof
TW202346599A (zh) 2022-02-08 2023-12-01 美商航海家醫療公司 Aav衣殼變異體及其用途
GB202201713D0 (en) 2022-02-10 2022-03-30 Univ Dundee An affinity directed phosphatase system for targeted protein dephosphorylation
WO2023187728A1 (en) 2022-04-01 2023-10-05 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2023212683A1 (en) 2022-04-29 2023-11-02 Kriya Therapeutics, Inc. Insulin and glucokinase gene therapy compositions and its use for treating diabetes
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
WO2023235791A1 (en) 2022-06-02 2023-12-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2024006741A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
WO2024042485A1 (en) 2022-08-25 2024-02-29 Takeda Pharmaceutical Company Limited Composition for use in the treatment of fabry disease
WO2024052413A1 (en) 2022-09-07 2024-03-14 Universitat Autònoma De Barcelona Beta-hexosaminidase vectors
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024054993A1 (en) 2022-09-09 2024-03-14 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic anti-ctla4 antibodies and methods of using the same
WO2024059739A1 (en) 2022-09-15 2024-03-21 Voyager Therapeutics, Inc. Tau binding compounds
WO2024056902A2 (en) 2022-09-16 2024-03-21 Christopher Shaw Compositions and methods for treating neurological diseases
WO2024064863A2 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors
WO2024064856A1 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of cardiomyopathy with aav gene therapy vectors

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) * 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
WO1991019803A1 (en) * 1990-06-19 1991-12-26 Applied Biotechnology, Incorporated Self assembled, defective, nonself-propagating viral particles
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

Also Published As

Publication number Publication date
EP0766569B1 (de) 2006-01-11
US5863541A (en) 1999-01-26
EP0766569A1 (de) 1997-04-09
AU705564B2 (en) 1999-05-27
CA2193802A1 (en) 1996-01-11
US6204059B1 (en) 2001-03-20
EP0766569A4 (de) 1999-06-02
JPH10502526A (ja) 1998-03-10
DE69534746D1 (de) 2006-04-06
US20010016355A1 (en) 2001-08-23
WO1996000587A1 (en) 1996-01-11
DE69534746T2 (de) 2006-09-14
AU2913895A (en) 1996-01-25

Similar Documents

Publication Publication Date Title
ATE315637T1 (de) Aav-capsidvehikel für den molekularen transport
FR17C1046I1 (fr) Nouvelles molecules b7-4 et leurs utilisations
ATE214100T1 (de) Herstellung von rekombinanten proteinen in einem rinderadenovirus-expressionsvektorsystem
HK1024714A1 (en) Dumbbell expression constructs for gene therapy
DE60035517D1 (de) Gl50 moleküle, sowie verwendungen derselben
BR9708387A (pt) Processo para vacinação de um mamìfero a um antìgeno selecionado, células patogênica e hospedeira, composição de vacina, ácido nucleico, e, opsonina engenheirada.
WO2000073316A3 (en) Virus immunologic determinants
PL409838A1 (pl) Rekombinowane wirusy stowarzyszone z adenowirusem (AAV), kompozycje , wyizolowane białko kapsydu, wyizolowana lub syntetyczna cząsteczka kwasu nukleinowego, sposób wytwarzania rekombinowanych wirusów, komórki gospodarza, białka obejmujące fragment białka kapsydu AAV, sztuczne białka, rekombinowane wirusy, cząsteczki, sposoby dostarczania transgenu do komórki , sposób identyfikacji serotypu sekwencji wirusa (AAV), zestaw diagnostyczny, sposób izolacji nowych wirusów, nowe serotypy wirusa, wyizolowane wirusy, rekombinowana komórka, zastosowanie wirusa
EP0460076A4 (en) Genetically engineered immunoglobulins
ATE465266T1 (de) Zusammensetzungen und verfahren zur verpackung von alpavirus-vektoren
ES2170622A1 (es) Clones y vectores infectivos derivados de coronavirus y sus aplicaciones.
IL77694A (en) Canine parvovirus vaccines comprising viruses of the canine parvovirus strain 154 or viruses derived therefrom by additional cell culture passaging
ATE315658T1 (de) Genkonstrukte für genimmunisierung
DE60041243D1 (de) Gebrauchsverfaheren eines neuen lysyloxidase-verwandten proteins
Beyer et al. Recombinant expression of lymphocytic choriomeningitis virus strain WE glycoproteins: a single amino acid makes the difference
Rueda et al. Influence of flanking sequences on presentation efficiency of a CD8+ cytotoxic T-cell epitope delivered by parvovirus-like particles
WO2001096392A3 (en) 22109, a novel human thioredoxin family member and uses thereof
WO2001073028A3 (en) A p-selectin glycoprotein ligand (psgl-1) binding protein and uses therefor
ATE435915T1 (de) Spezifische nukleinsäuren und polypeptide aus krankheitserregenden stämmen von neisseria
WO2002026803A3 (en) 22108 and 47916, novel human thioredoxin family members and uses thereof
Tikhonenko Prospects and achievements in the development of genetically engineered antiviral vaccines.
Reeves et al. Expression of rotavirus VP7 antigens in fusions with bacterial proteins
DE60124910D1 (de) Menschliche twik-8 moleküle und ihre verwendung
AU2001285204A1 (en) Atcr-1, a human acyltransferase and uses thereof
FI963016A (fi) Heterologisten proteiinien ilmentäminen heikennetyissä bakteereissa käyttämällä htrA-promoottoreita

Legal Events

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