ATE269412T1 - Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie - Google Patents

Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie

Info

Publication number
ATE269412T1
ATE269412T1 AT99918810T AT99918810T ATE269412T1 AT E269412 T1 ATE269412 T1 AT E269412T1 AT 99918810 T AT99918810 T AT 99918810T AT 99918810 T AT99918810 T AT 99918810T AT E269412 T1 ATE269412 T1 AT E269412T1
Authority
AT
Austria
Prior art keywords
gene therapy
methods
antitrypsin
viral vector
vector encoding
Prior art date
Application number
AT99918810T
Other languages
English (en)
Inventor
Terence R Flotte
Sihong Song
Barry J Byrne
Michael Morgan
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
Priority claimed from PCT/US1999/008921 external-priority patent/WO1999055564A2/en
Application granted granted Critical
Publication of ATE269412T1 publication Critical patent/ATE269412T1/de

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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Diabetes (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
AT99918810T 1998-04-24 1999-04-23 Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie ATE269412T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US8302598P 1998-04-24 1998-04-24
PCT/US1999/008921 WO1999055564A2 (en) 1998-04-24 1999-04-23 Materials and methods for gene therapy

Publications (1)

Publication Number Publication Date
ATE269412T1 true ATE269412T1 (de) 2004-07-15

Family

ID=22175690

Family Applications (1)

Application Number Title Priority Date Filing Date
AT99918810T ATE269412T1 (de) 1998-04-24 1999-04-23 Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie

Country Status (10)

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

Families Citing this family (23)

* 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 (de) * 2001-05-24 2009-04-15 Genzyme Corporation Muskelspezifische expressionsvektoren
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
ES2579231T3 (es) * 2008-01-15 2016-08-08 Abbvie Inc Vectores de expresión de mamíferos mejorados y usos de los mismos
US9375491B2 (en) 2011-10-28 2016-06-28 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.
EP3253406B1 (de) 2015-02-05 2023-11-01 Canem Holdings, LLC Zusammensetzungen zur behandlung von granulomatose mit polyangiitis
CA2979120A1 (en) 2015-03-03 2016-09-09 Fondazione Telethon Multiple aav vector system for expressing genes over 5 kilobases, comprising at least one degradation signal and uses thereof
AU2016317726B2 (en) 2015-08-28 2022-05-26 Serpin Pharma, Llc Methods for treatment of diseases
WO2017055370A1 (en) 2015-09-28 2017-04-06 Fondazione Telethon Treatment of bone growth disorders
ES2984415T3 (es) * 2017-12-12 2024-10-29 Kamada Ltd Métodos para inducir tolerancia inmunitaria y reducir la respuesta de anticuerpos anti-fármaco
JP7590962B2 (ja) 2018-11-05 2024-11-27 アレン インスティテュート 皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物
CA3142948A1 (en) 2019-02-15 2020-08-20 Allen Institute Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations
WO2021207077A1 (en) * 2020-04-06 2021-10-14 Homology Medicines, Inc. Adeno-associated virus compositions for ids gene transfer and methods of use thereof
WO2023213817A1 (en) 2022-05-02 2023-11-09 Fondazione Telethon Ets Gene therapy for gyrate atrophy of the choroid and retina
WO2024036250A2 (en) * 2022-08-10 2024-02-15 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof
IT202300004443A1 (it) 2023-03-09 2024-09-09 Int Centre For Genetic Engineering And Biotechnology Icgeb Sequenza codificante per alfa galattosidasi a umana per il trattamento della malattia di fabry
EP4724470A1 (de) 2023-06-12 2026-04-15 Children's Hospital Medical Center Aav-cftr-vektoren und verfahren zur verwendung davon
CN117223676B (zh) * 2023-09-25 2024-08-20 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
EP0733103B1 (de) 1993-11-09 2004-03-03 Targeted Genetics Corporation 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
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
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
WO1999055564A8 (en) 1999-12-02
DE69918090D1 (de) 2004-07-22
EP1071806A2 (de) 2001-01-31
WO1999055564A1 (en) 1999-11-04
CA2326327A1 (en) 1999-11-04
PT1071806E (pt) 2004-09-30
WO1999055564A9 (en) 2000-04-06
HK1035205A1 (en) 2001-11-16
DK1071806T3 (da) 2004-07-19
US20090186002A1 (en) 2009-07-23
NZ507308A (en) 2003-01-31
DE69918090T2 (de) 2005-06-16
ES2219011T3 (es) 2004-11-16
AU3663699A (en) 1999-11-16
EP1071806B1 (de) 2004-06-16
US20030082162A1 (en) 2003-05-01
US6461606B1 (en) 2002-10-08
CA2326327C (en) 2009-06-16
US20030095949A1 (en) 2003-05-22

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