DK1071806T3 - Rekombinant adeno-associeret virus-vektor, der koder for alpha-1-antitrypsin i genterapi - Google Patents

Rekombinant adeno-associeret virus-vektor, der koder for alpha-1-antitrypsin i genterapi

Info

Publication number
DK1071806T3
DK1071806T3 DK99918810T DK99918810T DK1071806T3 DK 1071806 T3 DK1071806 T3 DK 1071806T3 DK 99918810 T DK99918810 T DK 99918810T DK 99918810 T DK99918810 T DK 99918810T DK 1071806 T3 DK1071806 T3 DK 1071806T3
Authority
DK
Denmark
Prior art keywords
gene therapy
methods
antitrypsin
associated virus
vector encoding
Prior art date
Application number
DK99918810T
Other languages
English (en)
Inventor
Terence R Flotte
Sihong Song
Michael Morgan
Barry J Byrne
Original Assignee
Univ Florida
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 Florida filed Critical Univ Florida
Application granted granted Critical
Publication of DK1071806T3 publication Critical patent/DK1071806T3/da

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121—Serpins
    • C07K14/8125—Alpha-1-antitrypsin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/55—Protease inhibitors
    • A61K38/57—Protease inhibitors from animals; from humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
DK99918810T 1998-04-24 1999-04-23 Rekombinant adeno-associeret virus-vektor, der koder for alpha-1-antitrypsin i genterapi DK1071806T3 (da)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US8302598P 1998-04-24 1998-04-24

Publications (1)

Publication Number Publication Date
DK1071806T3 true DK1071806T3 (da) 2004-07-19

Family

ID=22175690

Family Applications (1)

Application Number Title Priority Date Filing Date
DK99918810T DK1071806T3 (da) 1998-04-24 1999-04-23 Rekombinant adeno-associeret virus-vektor, der koder for alpha-1-antitrypsin i genterapi

Country Status (11)

Country Link
US (4) US6461606B1 (da)
EP (1) EP1071806B1 (da)
AT (1) ATE269412T1 (da)
AU (1) AU3663699A (da)
CA (1) CA2326327C (da)
DE (1) DE69918090T2 (da)
DK (1) DK1071806T3 (da)
ES (1) ES2219011T3 (da)
HK (1) HK1035205A1 (da)
NZ (1) NZ507308A (da)
PT (1) PT1071806E (da)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US6821775B1 (en) * 2000-02-11 2004-11-23 Genvec, Inc. Viral vector encoding pigment epithelium-derived factor
CN1293196C (zh) 2000-05-26 2007-01-03 大日本住友制药株式会社 具有副作用降低的特性的新型重组腺病毒载体
EP1390490B1 (en) * 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
US7312202B2 (en) * 2003-02-18 2007-12-25 Board Of Regents, The University Of Texas System Rationally designed and chemically synthesized promoter for genetic vaccine and gene therapy
ES2247942B1 (es) * 2004-08-27 2006-10-01 Instituto Nacional De Investigacion Y Tecnologia Agraria Y Alimentaria (Inia) Construccion genica, vector y vacuna adn para la vacunacion de animales acuaticos.
US7479550B2 (en) * 2006-06-02 2009-01-20 The Board Of Regents Of The University Of Texas System Amyloid β gene vaccines
KR20100113112A (ko) * 2008-01-15 2010-10-20 아보트 러보러터리즈 개선된 포유동물 발현 벡터 및 이의 용도
WO2013063601A1 (en) 2011-10-28 2013-05-02 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
MX365403B (es) 2012-01-09 2019-05-31 Serpin Pharma Llc Peptidos y metodos para usarlos.
DK3253406T3 (da) 2015-02-05 2024-01-22 Canem Holdings Llc Sammensætninger til behandling af granulomatose med polyangiitis
EP3265571B1 (en) 2015-03-03 2022-04-13 Fondazione Telethon Multiple vector system and uses thereof
AU2016317726B2 (en) 2015-08-28 2022-05-26 Serpin Pharma, Llc Methods for treatment of diseases
CA2998267A1 (en) 2015-09-28 2017-04-06 Fondazione Telethon Treatment of bone growth disorders
EP3723790B1 (en) * 2017-12-12 2024-03-13 Kamada Ltd Methods of inducing immune tolerance and reducing anti-drug antibody response
US20220249703A1 (en) * 2019-02-15 2022-08-11 Allen Institute Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations
KR20220164743A (ko) * 2020-04-06 2022-12-13 호몰로지 메디슨, 인크. Ids 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
WO2023213817A1 (en) 2022-05-02 2023-11-09 Fondazione Telethon Ets Gene therapy for gyrate atrophy of the choroid and retina
CN117223676A (zh) * 2023-09-25 2023-12-15 武汉大学 面中部发育畸形动物选育方法、辅助选育试剂和预防药物

Family Cites Families (14)

* 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
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US6261834B1 (en) * 1991-11-08 2001-07-17 Research Corporation Technologies, Inc. Vector for gene therapy
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
US5439824A (en) * 1993-05-06 1995-08-08 The United States Of America Increased expression of α-1-antitrypsin in expression vectors through the inclusion of intron II
DE69433592T2 (de) 1993-11-09 2005-02-10 Targeted Genetics Corp., Seattle Die erzielung hoher titer des rekombinanten aav-vektors
US5693531A (en) * 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) * 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5652224A (en) * 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US6723707B1 (en) * 1997-08-25 2004-04-20 The Trustees Of The University Of Pennsylvania Use of insulin-like growth factor-I in muscle

Also Published As

Publication number Publication date
ES2219011T3 (es) 2004-11-16
EP1071806A2 (en) 2001-01-31
WO1999055564A8 (en) 1999-12-02
WO1999055564A1 (en) 1999-11-04
US6461606B1 (en) 2002-10-08
CA2326327A1 (en) 1999-11-04
EP1071806B1 (en) 2004-06-16
PT1071806E (pt) 2004-09-30
CA2326327C (en) 2009-06-16
DE69918090T2 (de) 2005-06-16
AU3663699A (en) 1999-11-16
WO1999055564A9 (en) 2000-04-06
DE69918090D1 (de) 2004-07-22
US20030082162A1 (en) 2003-05-01
US20090186002A1 (en) 2009-07-23
US20030095949A1 (en) 2003-05-22
NZ507308A (en) 2003-01-31
HK1035205A1 (en) 2001-11-16
ATE269412T1 (de) 2004-07-15

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