CA2269661A1 - Cassettes d'encapsidation d'aav activables par la recombinase servant a la production de vecteurs d'aav - Google Patents

Cassettes d'encapsidation d'aav activables par la recombinase servant a la production de vecteurs d'aav Download PDF

Info

Publication number
CA2269661A1
CA2269661A1 CA002269661A CA2269661A CA2269661A1 CA 2269661 A1 CA2269661 A1 CA 2269661A1 CA 002269661 A CA002269661 A CA 002269661A CA 2269661 A CA2269661 A CA 2269661A CA 2269661 A1 CA2269661 A1 CA 2269661A1
Authority
CA
Canada
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
CA002269661A
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 Corporation
Haim Burstein
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 Corporation, Haim Burstein filed Critical Targeted Genetics Corporation
Publication of CA2269661A1 publication Critical patent/CA2269661A1/fr
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
CA002269661A 1996-12-18 1997-12-12 Cassettes d'encapsidation d'aav activables par la recombinase servant a la production de vecteurs d'aav Abandoned CA2269661A1 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US4168996P 1996-12-18 1996-12-18
US60/041,689 1996-12-18
PCT/US1997/023018 WO1998027207A1 (fr) 1996-12-18 1997-12-12 Cassettes d'encapsidation d'aav activables par la recombinase servant a la production de vecteurs d'aav

Publications (1)

Publication Number Publication Date
CA2269661A1 true CA2269661A1 (fr) 1998-06-25

Family

ID=21917821

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002269661A Abandoned CA2269661A1 (fr) 1996-12-18 1997-12-12 Cassettes d'encapsidation d'aav activables par la recombinase servant a la production de vecteurs d'aav

Country Status (5)

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

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
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released 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
US6416992B1 (en) 1998-10-13 2002-07-09 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 (fr) 1999-05-28 2008-07-02 Targeted Genetics Corporation Procédés et compositions pour diminuer le niveau de facteur de nécrose de tumeur (TNF) pour des troubles associés au TNF
US6537540B1 (en) 1999-05-28 2003-03-25 Targeted Genetics Corporation Methods and composition for lowering the level of tumor necrosis factor (TNF) in TNF-associated disorders
EP1916258B1 (fr) 1999-08-09 2014-04-23 Targeted Genetics Corporation Améliorations de l'expression d'une séquence de nucléotides hétérologues à brin unique à partir de vecteurs viraux recombinants par la désignation de la séquence de manière à ce qu'elle forme des paires de bases intrabrins
DE60039766D1 (de) 1999-08-09 2008-09-18 Targeted Genetics Corp Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
DE19941186A1 (de) * 1999-08-30 2001-03-01 Peter Droege Sequenz-spezifische DNA-Rekombination in eukaryotischen Zellen
EP1134287A1 (fr) * 2000-03-08 2001-09-19 Universite De Geneve Système pour contrôler l'expression d'un gène donné en utilisant un autre gène codant pour un polypeptide avec une activité recombinante
JP2004536572A (ja) 2001-02-23 2004-12-09 セル・ジェネシス・インコーポレイテッド 新規ベクター構築物
NL1017852C2 (nl) * 2001-04-17 2002-10-29 Multigen Holding S A Recombinant nucleotidevolgorde welke codeert voor een gen.
EP1587923B1 (fr) 2003-01-22 2011-08-24 Duke University Constructions ameliorees permettant d'exprimer des polypeptides lysosomiaux
JP2007521835A (ja) 2004-02-10 2007-08-09 トラスティーズ・オブ・ダートマウス・カレッジ ニコチンアミドリボシドキナーゼ組成物およびそれらの使用方法
MX2011010097A (es) 2009-03-27 2011-10-19 Proyecto Biomedicina Cima Sl Metodos y composiciones para el tratamiento de cirrosis y fibrosis hepatica.
KR101835490B1 (ko) 2009-05-02 2018-03-08 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
WO2012145509A2 (fr) 2011-04-19 2012-10-26 The Research Foundation Of State University Of New York Séquences rep de virus adéno-associé, vecteurs, et virus
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
EP2981612B1 (fr) 2013-04-04 2019-07-03 Trustees of Dartmouth College Compositions et procédés pour l'excision in vivo d'adn proviral de vih-1
WO2017081204A1 (fr) * 2015-11-11 2017-05-18 Targeted Transgenesis Limited Expression de transgène sélective d'un tissu
CN110914413A (zh) 2017-02-17 2020-03-24 隆萨有限公司 产生腺相关病毒的哺乳动物细胞
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
MX2021010149A (es) 2019-02-25 2021-09-14 Novartis Ag Composiciones y metodos para tratar la distrofia cristalina de bietti.
EP3931335A2 (fr) 2019-02-25 2022-01-05 Novartis AG Compositions et procédés pour traiter une dystrophie cristalline de bietti
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
CA3197726A1 (fr) 2020-10-15 2022-04-21 Simon Auslaender Constructions d'acides nucleiques ameliorees pour activation de genes simultanee

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0796339A1 (fr) * 1994-12-06 1997-09-24 Targeted Genetics Corporation Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants
DE69631480T2 (de) * 1995-08-03 2004-11-25 Avigen Inc., Alameda Helfersystem zur wirkungssteigerung der aav-vektorproduktion
EP0950111A1 (fr) * 1996-09-06 1999-10-20 The Trustees Of The University Of Pennsylvania Procede d'utilisation de cre-lox pour la production de virus adeno-associes de recombinaison

Also Published As

Publication number Publication date
EP0946723A1 (fr) 1999-10-06
AU5603998A (en) 1998-07-15
JP2001506132A (ja) 2001-05-15
WO1998027207A1 (fr) 1998-06-25

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
CA2269661A1 (fr) Cassettes d'encapsidation d'aav activables par la recombinase servant a la production de vecteurs d'aav
US6642051B1 (en) Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
AU688428B2 (en) Generation of high titers of recombinant AAV vectors
US20210010027A1 (en) Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
US6924128B2 (en) Packaging cell lines for generation of high titers of recombinant AAV vectors
AU707866B2 (en) Packaging cell lines for generation of high titers of recombinant AAV vectors
WO1998027204A9 (fr) Genes d'encapsidation fractionnes de virus adeno-associe (aav) et lignees cellulaires comprenant ces genes utilises pour la production de vecteurs d'aav de recombinaison
US6936466B2 (en) Transcriptionally-activated AAV inverted terminal repeats (ITRs) for use with recombinant AAV vectors
US20040087026A1 (en) Host cells for packing a recombinant adeno-associated virus (raav), method for the production and use thereof
JP2006223315A (ja) Ａａｖベクター産生のための高効率ヘルパー系
WO1996017947A9 (fr) Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants
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

Legal Events

Date Code Title Description
FZDE Discontinued