AU5603998A - Recombinase-activatable AAV packaging cassettes for use in the production of AV vectors - Google Patents

Recombinase-activatable AAV packaging cassettes for use in the production of AV vectors

Info

Publication number
AU5603998A
AU5603998A AU56039/98A AU5603998A AU5603998A AU 5603998 A AU5603998 A AU 5603998A AU 56039/98 A AU56039/98 A AU 56039/98A AU 5603998 A AU5603998 A AU 5603998A AU 5603998 A AU5603998 A AU 5603998A
Authority
AU
Australia
Prior art keywords
aav
gene
recombinase
packaging
ssr
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
AU56039/98A
Other languages
English (en)
Inventor
Haim Burstein
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.)
Ampliphi Biosciences Corp
Original Assignee
Targeted Genetics Corp
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 Targeted Genetics Corp filed Critical Targeted Genetics Corp
Publication of AU5603998A publication Critical patent/AU5603998A/en
Abandoned legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
AU56039/98A 1996-12-18 1997-12-12 Recombinase-activatable AAV packaging cassettes for use in the production of AV vectors Abandoned AU5603998A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US4168996P 1996-12-18 1996-12-18
US60041689 1996-12-18
PCT/US1997/023018 WO1998027207A1 (en) 1996-12-18 1997-12-12 Recombinase-activatable aav packaging cassettes for use in the production of aav vectors

Publications (1)

Publication Number Publication Date
AU5603998A true AU5603998A (en) 1998-07-15

Family

ID=21917821

Family Applications (1)

Application Number Title Priority Date Filing Date
AU56039/98A Abandoned AU5603998A (en) 1996-12-18 1997-12-12 Recombinase-activatable AAV packaging cassettes for use in the production of AV vectors

Country Status (5)

Country Link
EP (1) EP0946723A1 (ja)
JP (1) JP2001506132A (ja)
AU (1) AU5603998A (ja)
CA (1) CA2269661A1 (ja)
WO (1) WO1998027207A1 (ja)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO2000022152A1 (en) * 1998-10-13 2000-04-20 Avigen, Inc. Compositions and methods for producing recombinant adeno-associated virus
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
EP1939300A1 (en) 1999-05-28 2008-07-02 Targeted Genetics Corporation Methods and compositions for lowering the level of tumor necrosis factor (TNF) in TNF-associated disorders
ES2312344T3 (es) 1999-05-28 2009-03-01 Targeted Genetics Corporation Metodos y composiciones para disminuir el nivel de factor de necrosis tumoral (tnf) en trastornos asociados con tnf.
EP1916258B1 (en) 1999-08-09 2014-04-23 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
DE19941186A1 (de) * 1999-08-30 2001-03-01 Peter Droege Sequenz-spezifische DNA-Rekombination in eukaryotischen Zellen
EP1134287A1 (en) * 2000-03-08 2001-09-19 Universite De Geneve A system to control the expression of a given gene using another gene that encodes a polypeptide with recombinant activity
US7109029B2 (en) 2001-02-23 2006-09-19 Cell Genesys, Inc. Vector constructs
NL1017852C2 (nl) * 2001-04-17 2002-10-29 Multigen Holding S A Recombinant nucleotidevolgorde welke codeert voor een gen.
ES2371913T3 (es) 2003-01-22 2012-01-11 Duke University Constructos mejorados para expresar polipéptidos lisosomales.
EP1723227A4 (en) 2004-02-10 2007-09-19 Dartmouth College NICOTINAMIDRIBOSIDE KINASE COMPOSITIONS AND METHOD FOR THEIR USE
MX2011010097A (es) 2009-03-27 2011-10-19 Proyecto Biomedicina Cima Sl Metodos y composiciones para el tratamiento de cirrosis y fibrosis hepatica.
KR20180027619A (ko) 2009-05-02 2018-03-14 젠자임 코포레이션 신경변성 질환에 대한 유전자 요법
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
US20140155469A1 (en) 2011-04-19 2014-06-05 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors and viruses
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
CA2908253C (en) 2013-04-04 2024-01-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
WO2017081204A1 (en) * 2015-11-11 2017-05-18 Targeted Transgenesis Limited Tissue selective transgene expression
JP2020507331A (ja) 2017-02-17 2020-03-12 ロンザ リミテッドＬｏｎｚａ Ｌｉｍｉｔｅｄ アデノ随伴ウイルスを生産するための哺乳動物細胞
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
EP3931334A1 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy
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
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
KR20230085170A (ko) 2020-10-15 2023-06-13 에프. 호프만-라 로슈 아게 동시 유전자 활성화를 위한 핵산 구조체

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU707866B2 (en) * 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
ATE258987T1 (de) * 1995-08-03 2004-02-15 Avigen Inc Helfersystem zur wirkungssteigerung der aav- vektorproduktion
CA2264499A1 (en) * 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses

Also Published As

Publication number Publication date
JP2001506132A (ja) 2001-05-15
CA2269661A1 (en) 1998-06-25
WO1998027207A1 (en) 1998-06-25
EP0946723A1 (en) 1999-10-06

Similar Documents

Publication Publication Date Title
US6541258B2 (en) AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
AU5603998A (en) Recombinase-activatable AAV packaging cassettes for use in the production of AV vectors
US6642051B1 (en) Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
WO1998027204A9 (en) Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
AU707866B2 (en) Packaging cell lines for generation of high titers of recombinant AAV vectors
US6936466B2 (en) Transcriptionally-activated AAV inverted terminal repeats (ITRs) for use with recombinant AAV vectors
AU688428B2 (en) Generation of high titers of recombinant AAV vectors
US6924128B2 (en) Packaging cell lines for generation of high titers of recombinant AAV vectors
US6893865B1 (en) Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
US20040087026A1 (en) Host cells for packing a recombinant adeno-associated virus (raav), method for the production and use thereof
WO1996017947A9 (en) Packaging cell lines for generation of high titers of recombinant aav vectors
AU749352B2 (en) A conditional replication and expression system
AU758541B2 (en) Amplifiable adeno-associated virus (AAV) packaging cassettes for the production of recombinant AAV vectors
US7208315B2 (en) Compositions and methods for efficient AAV vector production
EP1025243A2 (en) TRANSCRIPTIONALLY-ACTIVATED AAV INVERTED TERMINAL REPEATS (ITRs) FOR USE WITH RECOMBINANT AAV VECTORS
AU2003203790B2 (en) Transcriptionally-activated AAV inverted terminal repeats (ITRs) for use with recombinant AAV vectors

Legal Events

Date Code Title Description
MK5 Application lapsed section 142(2)(e) - patent request and compl. specification not accepted