PT1071806E - Vector viral adeno-associado recombinante que codifica a alfa-1-antitripsina para terapia genetica - Google Patents

Vector viral adeno-associado recombinante que codifica a alfa-1-antitripsina para terapia genetica

Info

Publication number
PT1071806E
PT1071806E PT99918810T PT99918810T PT1071806E PT 1071806 E PT1071806 E PT 1071806E PT 99918810 T PT99918810 T PT 99918810T PT 99918810 T PT99918810 T PT 99918810T PT 1071806 E PT1071806 E PT 1071806E
Authority
PT
Portugal
Prior art keywords
methods
antitrypsin
aden
recombinant
viral vector
Prior art date
Application number
PT99918810T
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
Publication of PT1071806E publication Critical patent/PT1071806E/pt

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)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Diabetes (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Endocrinology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Emergency Medicine (AREA)
  • Chemical Kinetics & Catalysis (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)
PT99918810T 1998-04-24 1999-04-23 Vector viral adeno-associado recombinante que codifica a alfa-1-antitripsina para terapia genetica PT1071806E (pt)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
PT1071806E true PT1071806E (pt) 2004-09-30

Family

ID=22175690

Family Applications (1)

Application Number Title Priority Date Filing Date
PT99918810T PT1071806E (pt) 1998-04-24 1999-04-23 Vector viral adeno-associado recombinante que codifica a alfa-1-antitripsina para terapia genetica

Country Status (12)

Country Link
US (4) US6461606B1 (pt)
EP (1) EP1071806B1 (pt)
AT (1) ATE269412T1 (pt)
AU (1) AU3663699A (pt)
CA (1) CA2326327C (pt)
DE (1) DE69918090T2 (pt)
DK (1) DK1071806T3 (pt)
ES (1) ES2219011T3 (pt)
HK (1) HK1035205A1 (pt)
NZ (1) NZ507308A (pt)
PT (1) PT1071806E (pt)
WO (1) WO1999055564A1 (pt)

Families Citing this family (25)

* 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
EP1226752A4 (en) * 1999-11-05 2004-05-06 Kyowa Hakko Kogyo Kk NON-HUMAN TRANSGENIC MAMMALS FOR CONTROLLING CHANGE IN CALCIUM ION CONCENTRATION IN CELLS
US6821775B1 (en) * 2000-02-11 2004-11-23 Genvec, Inc. Viral vector encoding pigment epithelium-derived factor
KR20030072214A (ko) 2000-05-26 2003-09-13 스미또모 세이야꾸 가부시키가이샤 완화된 부작용을 갖는 신규 재조합 아데노바이러스 벡터
DE60231975D1 (de) * 2001-05-24 2009-05-28 Genzyme Corp Muskelspezifische expressionsvektoren
WO2003089011A1 (en) * 2002-04-19 2003-10-30 University Of Florida rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES
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
EP2240581B1 (en) * 2008-01-15 2016-05-11 AbbVie Inc. Improved mammalian expression vectors and uses thereof
AU2012327236B2 (en) 2011-10-28 2016-11-10 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
AU2013208293B2 (en) 2012-01-09 2017-08-10 Serpin Pharma, Llc Peptides and methods of using same
US11446366B2 (en) 2015-02-05 2022-09-20 Canem Holdings, Llc Compositions and methods for treating granulomatosis with polyangiitis
HRP20220776T1 (hr) 2015-03-03 2022-09-16 Fondazione Telethon Sustav višestrukih vektora i njegova upotreba
CA2996975C (en) 2015-08-28 2022-02-15 Serpin Pharma, Llc Methods for treatment of diseases
CN108431033A (zh) 2015-09-28 2018-08-21 泰莱托恩基金会 骨生长病症的治疗
IL275328B2 (en) * 2017-12-12 2024-03-01 Kamada Ltd Methods for inducing immune tolerance and reducing antibody response against drugs
EA202091844A1 (ru) * 2018-02-01 2021-02-03 Хомолоджи Медисинс, Инк. Композиции аденоассоциированных вирусов для переноса гена pah и способы их применения
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF
CA3142948A1 (en) * 2019-02-15 2020-08-20 Allen Institute Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations
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
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
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 (16)

* 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
JPH09509564A (ja) 1993-11-09 1997-09-30 ターゲテッド ジェネティックス コーポレイション 高力価組換えａａｖベクターの生成
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
US5780447A (en) * 1996-06-14 1998-07-14 St. Jude Children's Research Hospital Recombinant adeno-associated viral vectors
NZ336185A (en) * 1996-12-05 2001-02-23 Introgene Bv Genetic modification of primate hemopoietic repopulating stem cells
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
WO1999055564A9 (en) 2000-04-06
US6461606B1 (en) 2002-10-08
AU3663699A (en) 1999-11-16
ATE269412T1 (de) 2004-07-15
US20090186002A1 (en) 2009-07-23
EP1071806A2 (en) 2001-01-31
ES2219011T3 (es) 2004-11-16
WO1999055564A8 (en) 1999-12-02
DE69918090T2 (de) 2005-06-16
US20030082162A1 (en) 2003-05-01
NZ507308A (en) 2003-01-31
DK1071806T3 (da) 2004-07-19
DE69918090D1 (de) 2004-07-22
US20030095949A1 (en) 2003-05-22
WO1999055564A1 (en) 1999-11-04
HK1035205A1 (en) 2001-11-16
EP1071806B1 (en) 2004-06-16
CA2326327C (en) 2009-06-16
CA2326327A1 (en) 1999-11-04

Similar Documents

Publication Publication Date Title
PT1071806E (pt) Vector viral adeno-associado recombinante que codifica a alfa-1-antitripsina para terapia genetica
DK1019073T3 (da) Apoliprotein A-I agonister og deres anvendelse til at behandle dyslipidæmiske lidelser
DE69834936D1 (de) Vektor zur gewebespezifischen replikation und expression
HUP0003924A2 (hu) Génterápiás megközelítések ApoA-I agonisták biztosítására, valamint ezek alkalmazása diszlipidémiás rendellenességek kezelésében
DE69534791D1 (de) Vektoren zur gewebsspezifischen replikation
DE69528054T2 (de) In prostata aktiver gewebespezifischer enhancer
DE69635173D1 (de) Rekombinante MVA Viren und deren Verwendung
DE69703689T2 (de) Therapeutische anwendungen von bpi-protein-produkten zur behandlung von humaner meningococcämia
DK1262177T3 (da) Medicinsk brug af thyromimetiske forbindelser til behandling af hårtab og kompositioner
DE69636072D1 (de) Varianten des p53-proteins und deren therapeutischen verwendungen
TR200100348T2 (tr) Gen tedavisi teknikleri
CO5280032A1 (es) COMPOSICIONES RELACIONADAS CON ESTEAROIL-CoA DESATURASA HUMANA Y METODOS PARA TRATAR DESORDENES DE LA PIEL
DE60315628D1 (de) Modifizierte cea nucleinsäure und expressionsvektoren
ATE287722T1 (de) Mittel zur behandlung maligner erkrankungen unter verwendung des proteins yb-1
MXPA03008210A (es) Produccion de altos niveles del factor ix transgenico sin rescate del gen, y sus usos terapeuticos.
ATE246924T1 (de) Neue therapeutische verwendung von nicergoline
DE602005021159D1 (de) Bakteriophagen- und prophagen-proteine in der krebsgentherapie
DE69832883D1 (de) Transgene schweine
ATE364625T1 (de) Proteine aus yaba monkey tumor virus mit modulatorischer aktivität
NO964894L (no) Rekombinante viruser, deres fremstilling samt anvendelse av genterapi
ATE206617T1 (de) Verwendung von escherichia coli stamm dsm 6601 zur behandlung von diarrhoeen auf dem veterinärsektor
HUP9800319A2 (hu) Biostimulációs eljárás és berendezés
MX9707549A (es) Vectores virales y su uso para tratar trastornos hiperproliferativos, en particular restenosis.