AU2001245924A1 - Aav5 vector for transducing brain cells and lung cells - Google Patents

Aav5 vector for transducing brain cells and lung cells

Info

Publication number
AU2001245924A1
AU2001245924A1 AU2001245924A AU4592401A AU2001245924A1 AU 2001245924 A1 AU2001245924 A1 AU 2001245924A1 AU 2001245924 A AU2001245924 A AU 2001245924A AU 4592401 A AU4592401 A AU 4592401A AU 2001245924 A1 AU2001245924 A1 AU 2001245924A1
Authority
AU
Australia
Prior art keywords
cells
transducing
aav5 vector
brain
lung
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2001245924A
Inventor
John A. Chiorini
Beverly Davidson
Robert M. Kotin
Joseph Zabner
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Iowa Research Foundation UIRF
US Department of Health and Human Services
Original Assignee
University of Iowa Research Foundation UIRF
US Department of Health and Human Services
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 University of Iowa Research Foundation UIRF, US Department of Health and Human Services filed Critical University of Iowa Research Foundation UIRF
Publication of AU2001245924A1 publication Critical patent/AU2001245924A1/en
Abandoned legal-status Critical Current

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
    • 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
    • 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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2001245924A 2000-03-22 2001-03-22 Aav5 vector for transducing brain cells and lung cells Abandoned AU2001245924A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US09/533,427 US6855314B1 (en) 2000-03-22 2000-03-22 AAV5 vector for transducing brain cells and lung cells
US09533427 2000-03-22
PCT/US2001/009123 WO2001070276A2 (en) 2000-03-22 2001-03-22 Aav5 vector for transducing brain cells and lung cells

Publications (1)

Publication Number Publication Date
AU2001245924A1 true AU2001245924A1 (en) 2001-10-03

Family

ID=24125908

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001245924A Abandoned AU2001245924A1 (en) 2000-03-22 2001-03-22 Aav5 vector for transducing brain cells and lung cells

Country Status (3)

Country Link
US (1) US6855314B1 (en)
AU (1) AU2001245924A1 (en)
WO (1) WO2001070276A2 (en)

Families Citing this family (70)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
AU2001255575B2 (en) * 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US20050032219A1 (en) * 2001-07-03 2005-02-10 Patrick Aubourg Methods of administering vectors to synaptically connected neurons
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
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
PT1453547T (en) 2001-12-17 2016-12-28 Univ Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
WO2003093479A1 (en) * 2002-05-01 2003-11-13 University Of Florida Research Foundation, Inc. Improved 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
US7618948B2 (en) 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
US7994149B2 (en) 2003-02-03 2011-08-09 Medtronic, Inc. Method for treatment of Huntington's disease through intracranial delivery of sirna
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
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
JP2007528862A (en) * 2003-07-16 2007-10-18 アールブイエックス セラピューティクス, インコーポレイテッド Compounds and methods for downregulating the effects of TGF-β
WO2005038035A2 (en) * 2003-10-15 2005-04-28 University Of Iowa Research Foundation Methods for producing and using in vivo pseudotyped retroviruses
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
JP2008503590A (en) * 2004-06-21 2008-02-07 メドトロニック・インコーポレーテッド Medical systems and methods for delivering compositions to cells
US20080188431A1 (en) * 2004-09-08 2008-08-07 Chiorini John A Transcytosis of Adeno-Associated Viruses
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
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US9375440B2 (en) 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US20100076061A1 (en) * 2008-04-30 2010-03-25 University Of Iowa Research Foundation Repeated administration of lentiviral vectors to respiratory cells
US9133479B2 (en) * 2009-06-03 2015-09-15 Cedars-Sinai Medical Center Effective vector platform for gene transfer and gene therapy
WO2012007458A1 (en) 2010-07-12 2012-01-19 Universidad Autónoma De Barcelona Gene therapy composition for use in diabetes treatment
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP2691529B1 (en) 2011-03-31 2019-06-12 University of Iowa Research Foundation AAV2 particle comprising an AAV2 capsid protein and a vector comprising a nucleic acid encoding a tripeptidyl peptidase 1 (TPP1) for use in treating late infantile ceroid lipofuscinosis (LINCL) in a non-rodent mammal by intraventricular injection or ICV delivery.
JP6072772B2 (en) 2011-04-22 2017-02-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Adeno-associated virus virions with mutant capsids and methods of use thereof
CA2873890C (en) 2012-05-18 2022-12-06 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits
WO2014194132A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US20170336395A1 (en) 2013-11-20 2017-11-23 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits
US9890365B2 (en) 2014-03-09 2018-02-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency
IL259321B2 (en) 2014-03-17 2023-10-01 Univ Washington Compositions and methods for enhanced gene expression in cone cells
CN106460062A (en) 2014-05-05 2017-02-22 美敦力公司 Methods and compositions for SCD, CRT, CRT-D, or SCA therapy identification and/or selection
AU2015255877B2 (en) * 2014-05-08 2020-03-26 Chdi Foundation, Inc. Methods and compositions for treating huntington's disease
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3194430A1 (en) 2014-09-16 2017-07-26 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
CN107106689A (en) 2014-11-05 2017-08-29 沃雅戈治疗公司 AADC polynucleotides for treating Parkinson's
MX2017006216A (en) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als).
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
CN114480502A (en) 2015-03-02 2022-05-13 阿德夫拉姆生物技术股份有限公司 Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
JP6836999B2 (en) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California Adeno-associated virus mutants and how to use them
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
US11027024B2 (en) * 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
EP3384035A4 (en) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR102392236B1 (en) 2016-05-18 2022-05-03 보이저 테라퓨틱스, 인크. Regulatory Polynucleotides
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
CN114672516A (en) 2016-07-29 2022-06-28 加利福尼亚大学董事会 Adeno-associated virus virions with variant capsids and methods of use thereof
EP3831281A1 (en) 2016-08-30 2021-06-09 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
EP3528785A4 (en) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP3619308A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
JP7229989B2 (en) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド Trajectory array guide system
KR20200044793A (en) 2017-08-03 2020-04-29 보이저 테라퓨틱스, 인크. Compositions and methods for delivery of AAV
JP2020534788A (en) 2017-08-28 2020-12-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Adeno-associated virus capsid mutant and how to use it
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP7502991B2 (en) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド Treatment of amyotrophic lateral sclerosis (ALS)
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
CA3107462A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
US20210317478A1 (en) * 2018-08-21 2021-10-14 Massachusetts Eye And Ear Infirmary Compositions and methods for modulating transduction efficiency of adeno-associated viruses
EP3861113A1 (en) 2018-10-04 2021-08-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
SG11202103425YA (en) 2018-10-05 2021-05-28 Voyager Therapeutics Inc Engineered nucleic acid constructs encoding aav production proteins
EP3867389A1 (en) 2018-10-15 2021-08-25 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
EP4153760A4 (en) * 2020-05-21 2024-08-21 Nysnobio Gt Llc Gene therapy delivery of parkin mutants having increased activity to treat parkinson's disease
CN115044616B (en) * 2022-06-28 2024-05-17 中国科学院深圳先进技术研究院 Adeno-associated virus vector for analyzing brain single-stage output neural network and application thereof
WO2024055020A2 (en) * 2022-09-09 2024-03-14 The Regents Of The University Of California Reprogramming tropism via displayed peptides tiling receptor-ligands

Family Cites Families (12)

* 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
JPH10501686A (en) * 1994-04-13 1998-02-17 ザ ロックフェラー ユニヴァーシティ AAV-mediated delivery of DNA to cells of the nervous system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
DE4436664A1 (en) 1994-10-13 1996-07-04 Max Planck Gesellschaft Cells that stably express specific adeno-associated virus Rep proteins
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
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
JP4289687B2 (en) 1997-03-14 2009-07-01 ザ チルドレンズ ホスピタル オブ フィラデルフィア Methods and compositions for use in gene therapy for the treatment of hemophilia
IT1291135B1 (en) 1997-04-08 1998-12-29 Angeletti P Ist Richerche Bio RECOMBINANT VECTORS USABLE IN GENE THERAPY
CA2329259C (en) * 1998-05-27 2003-08-05 Avigen, Inc. Convection-enhanced delivery of aav vectors
WO1999061601A2 (en) * 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells

Also Published As

Publication number Publication date
WO2001070276A3 (en) 2002-06-27
WO2001070276A2 (en) 2001-09-27
US6855314B1 (en) 2005-02-15

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