DE69334238D1 - Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn - Google Patents

Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn

Info

Publication number
DE69334238D1
DE69334238D1 DE69334238T DE69334238T DE69334238D1 DE 69334238 D1 DE69334238 D1 DE 69334238D1 DE 69334238 T DE69334238 T DE 69334238T DE 69334238 T DE69334238 T DE 69334238T DE 69334238 D1 DE69334238 D1 DE 69334238D1
Authority
DE
Germany
Prior art keywords
cells
nervous system
central nervous
adenovirus
brain
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.)
Expired - Lifetime
Application number
DE69334238T
Other languages
English (en)
Inventor
Axel Kahn
Jacques Mallet
Michel Perricaudet
Marc Peschanski
Jean-Jacques Robert
Gal La Salle Gildas Le
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.)
Institut National de la Sante et de la Recherche Medicale INSERM
Aventis Pharma SA
Original Assignee
Institut National de la Sante et de la Recherche Medicale INSERM
Aventis Pharma SA
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 Institut National de la Sante et de la Recherche Medicale INSERM, Aventis Pharma SA filed Critical Institut National de la Sante et de la Recherche Medicale INSERM
Application granted granted Critical
Publication of DE69334238D1 publication Critical patent/DE69334238D1/de
Anticipated expiration legal-status Critical
Expired - Lifetime 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
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0006—Oxidoreductases (1.) acting on CH-OH groups as donors (1.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16002—Tyrosine 3-monooxygenase (1.14.16.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01051—Alpha-L-fucosidase (3.2.1.51)
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Psychology (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
DE69334238T 1992-09-25 1993-09-17 Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn Expired - Lifetime DE69334238D1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP92402644 1992-09-25
PCT/EP1993/002519 WO1994008026A1 (en) 1992-09-25 1993-09-17 Adenovirus vectors for the transfer of foreign genes into cells of the central nervous system, particularly in brain

Publications (1)

Publication Number Publication Date
DE69334238D1 true DE69334238D1 (de) 2008-09-25

Family

ID=8211713

Family Applications (1)

Application Number Title Priority Date Filing Date
DE69334238T Expired - Lifetime DE69334238D1 (de) 1992-09-25 1993-09-17 Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn

Country Status (14)

Country Link
US (2) US6756523B1 (de)
EP (1) EP0669987B1 (de)
JP (1) JPH08501686A (de)
AT (1) ATE404683T1 (de)
AU (1) AU692423B2 (de)
CA (1) CA2145535C (de)
DE (1) DE69334238D1 (de)
ES (1) ES2312164T3 (de)
FI (1) FI951404A (de)
HU (1) HU219304B (de)
NO (1) NO951121D0 (de)
NZ (1) NZ256018A (de)
WO (1) WO1994008026A1 (de)
ZA (1) ZA937051B (de)

Families Citing this family (132)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6410010B1 (en) 1992-10-13 2002-06-25 Board Of Regents, The University Of Texas System Recombinant P53 adenovirus compositions
WO1992015680A1 (en) 1991-03-06 1992-09-17 Board Of Regents, The University Of Texas System Methods and compositions for the selective inhibition of gene expression
US5747469A (en) 1991-03-06 1998-05-05 Board Of Regents, The University Of Texas System Methods and compositions comprising DNA damaging agents and p53
FR2717496B1 (fr) * 1994-03-18 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
US6472178B1 (en) * 1998-02-27 2002-10-29 Regeneron Pharmaceuticals, Inc. Nucleic acids encoding a modified ciliary neurotrophic factor and method of making thereof
FR2705686B1 (fr) * 1993-05-28 1995-08-18 Transgene Sa Nouveaux adénovirus défectifs et lignées de complémentation correspondantes.
AU710962B2 (en) * 1993-05-28 1999-09-30 Transgene S.A. Defective adenoviruses and corresponding complementation lines
US5919676A (en) * 1993-06-24 1999-07-06 Advec, Inc. Adenoviral vector system comprising Cre-loxP recombination
US6140087A (en) * 1993-06-24 2000-10-31 Advec, Inc. Adenovirus vectors for gene therapy
US6080569A (en) * 1993-06-24 2000-06-27 Merck & Co., Inc. Adenovirus vectors generated from helper viruses and helper-dependent vectors
US7045347B2 (en) 1993-06-24 2006-05-16 Advec, Inc. Helper dependent adenovirus vectors based on integrase family site-specific recombinases
US6730507B1 (en) 1993-06-24 2004-05-04 Merck & Co., Inc. Use of helper-dependent adenoviral vectors of alternative serotypes permits repeat vector administration
US6120764A (en) * 1993-06-24 2000-09-19 Advec, Inc. Adenoviruses for control of gene expression
DE69434486T2 (de) * 1993-06-24 2006-07-06 Advec Inc. Adenovirus vektoren für gentherapie
US20020136708A1 (en) 1993-06-24 2002-09-26 Graham Frank L. System for production of helper dependent adenovirus vectors based on use of endonucleases
FR2716460B1 (fr) * 1994-02-21 2002-08-09 Rhone Poulenc Rorer Sa Modèle animal de la maladie d'Alzheimer, préparation et utilisations.
FR2717495B1 (fr) * 1994-03-18 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
FR2717497B1 (fr) * 1994-03-18 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
IL113052A0 (en) * 1994-03-23 1995-06-29 Rhone Poulenc Rorer Sa Recombinant viruses, their preparation and their use in gene therapy
FR2726575B1 (fr) * 1994-11-09 1996-12-20 Rhone Poulenc Rorer Sa Virus recombinants, preparation et utilisation en therapie genique
FR2717823B1 (fr) * 1994-03-23 1996-04-26 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
FR2717824B1 (fr) * 1994-03-25 1996-04-26 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
FR2718150B1 (fr) * 1994-03-29 1996-04-26 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
EP0763103A4 (de) * 1994-04-28 1998-07-15 Univ Michigan Genverabreichender vektor aus in adenvirus verpackter plasmid-dna und eine verpackungszellinie
US6379943B1 (en) 1999-03-05 2002-04-30 Merck & Co., Inc. High-efficiency Cre/loxp based system for construction of adenovirus vectors
DE69535178T2 (de) * 1994-06-10 2006-12-14 Genvec, Inc. Adenoviren-vektor systeme und zelllinien
US5851806A (en) * 1994-06-10 1998-12-22 Genvec, Inc. Complementary adenoviral systems and cell lines
GB9506466D0 (en) * 1994-08-26 1995-05-17 Prolifix Ltd Cell cycle regulated repressor and dna element
US5846782A (en) * 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
US6465253B1 (en) 1994-09-08 2002-10-15 Genvec, Inc. Vectors and methods for gene transfer to cells
JP3565859B2 (ja) 1994-10-28 2004-09-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 改良されたアデノウイルスおよびその使用法
FR2727867B1 (fr) * 1994-12-13 1997-01-31 Rhone Poulenc Rorer Sa Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux
US5780296A (en) * 1995-01-17 1998-07-14 Thomas Jefferson University Compositions and methods to promote homologous recombination in eukaryotic cells and organisms
US5770442A (en) * 1995-02-21 1998-06-23 Cornell Research Foundation, Inc. Chimeric adenoviral fiber protein and methods of using same
US6127525A (en) * 1995-02-21 2000-10-03 Cornell Research Foundation, Inc. Chimeric adenoviral coat protein and methods of using same
US6974694B2 (en) 1995-06-07 2005-12-13 Advec, Inc. Adenoviruses for control of gene expression
DE69638058D1 (de) 1995-06-15 2009-11-26 Crucell Holland Bv Verpackungssysteme für humane rekombinante Adenoviren zur Gentherapie
US6783980B2 (en) 1995-06-15 2004-08-31 Crucell Holland B.V. Packaging systems for human recombinant adenovirus to be used in gene therapy
AU7719596A (en) * 1995-11-07 1997-05-29 Baylor College Of Medicine Adenovirus-mediated production of bioactive proteins by mammalian cells and animals
US6132989A (en) * 1996-06-03 2000-10-17 University Of Washington Methods and compositions for enhanced stability of non-adenoviral DNA
FR2753379B1 (fr) * 1996-09-13 1998-10-30 Rhone Poulenc Rorer Sa Methode de traitement de la sclerose laterale amyotrophique
US6544523B1 (en) 1996-11-13 2003-04-08 Chiron Corporation Mutant forms of Fas ligand and uses thereof
US6696423B1 (en) 1997-08-29 2004-02-24 Biogen, Inc. Methods and compositions for therapies using genes encoding secreted proteins such as interferon-beta
BR9813930A (pt) 1997-11-06 2006-12-19 Chiron Spa antìgeno neisserial
AU2144899A (en) * 1998-01-13 1999-08-02 Claude Gravel Viral vectors expressing self-polymerizing neuronal intermediate filaments and their use
AU2144999A (en) * 1998-01-13 1999-08-02 Claude Gravel Viral vectors encoding neurofilament heavy proteins and their use
AU1956599A (en) * 1998-01-13 1999-08-02 Claude Gravel Viral vectors encoding neurofilament light proteins and their use
AU1979599A (en) 1998-01-14 1999-08-02 Chiron S.P.A. (neisseria meningitidis) antigens
EP2261347A3 (de) 1998-05-01 2012-01-11 Novartis Vaccines and Diagnostics, Inc. Antigene und Zusammensetzungen gegen Neisseria meningitidis
US20030017138A1 (en) 1998-07-08 2003-01-23 Menzo Havenga Chimeric adenoviruses
US6929946B1 (en) 1998-11-20 2005-08-16 Crucell Holland B.V. Gene delivery vectors provided with a tissue tropism for smooth muscle cells, and/or endothelial cells
CN100392082C (zh) 1999-04-30 2008-06-04 启龙股份公司 保守的奈瑟球菌抗原
US6913922B1 (en) 1999-05-18 2005-07-05 Crucell Holland B.V. Serotype of adenovirus and uses thereof
US6492169B1 (en) 1999-05-18 2002-12-10 Crucell Holland, B.V. Complementing cell lines
GB9911683D0 (en) 1999-05-19 1999-07-21 Chiron Spa Antigenic peptides
GB9916529D0 (en) 1999-07-14 1999-09-15 Chiron Spa Antigenic peptides
ES2541830T3 (es) 1999-10-29 2015-07-27 Novartis Vaccines And Diagnositics S.R.L. Péptidos antigénicos de Neisseria
PT1248647E (pt) 2000-01-17 2010-11-18 Novartis Vaccines & Diagnostics Srl Vacina de vesícula da membrana externa (omv) compreendendo proteínas de membrana externa de n. meningitidis do serogrupo b
JP2003530835A (ja) * 2000-04-06 2003-10-21 ケイオウエス ファーマシューティカルズ インコーポレイテッド 高トリグリセリド血症を誘発することなくコレステロールレベルを低下するための化合物と方法
US20020123093A1 (en) * 2000-04-06 2002-09-05 Zannis Vassilis I. Compounds and methods for lowering cholesterol levels without inducing hypertrigylceridemia
US7235233B2 (en) 2000-09-26 2007-06-26 Crucell Holland B.V. Serotype 5 adenoviral vectors with chimeric fibers for gene delivery in skeletal muscle cells or myoblasts
EP2277895B1 (de) 2000-10-27 2013-08-14 Novartis Vaccines and Diagnostics S.r.l. Nukleinsäure und Proteine von Gruppen A und B Streptokokken
GB0107658D0 (en) 2001-03-27 2001-05-16 Chiron Spa Streptococcus pneumoniae
GB0107661D0 (en) 2001-03-27 2001-05-16 Chiron Spa Staphylococcus aureus
FR2825372B1 (fr) * 2001-06-01 2004-06-18 Centre Nat Rech Scient Pseudotypage des vecteurs vih-1 par des enveloppes de rhabdovirus
US20050106162A1 (en) 2001-12-12 2005-05-19 Guido Grandi Immunisation against chlamydia trachomatis
US20030158112A1 (en) 2002-02-15 2003-08-21 Johns Hopkins University School Of Medicine Selective induction of apoptosis to treat ocular disease
EP1495121A2 (de) 2002-04-18 2005-01-12 Lynkeus Biotech GmbH Mitteln und verfahren zur spezifischen hemmung von genen in zellen und gewebe vom zns und/oder dem auge
IL164803A0 (en) 2002-04-25 2005-12-18 Crucell Holland Bv Means and methods for the production of adenovirusvectors
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
GB0308198D0 (en) 2003-04-09 2003-05-14 Chiron Srl ADP-ribosylating bacterial toxin
CA2534351C (en) * 2003-08-01 2018-10-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Accelerated vaccination
ES2407465T3 (es) 2004-03-29 2013-06-12 Galpharma Co., Ltd. Nueva proteína galectina 9 modificada y uso de la misma
US7309606B2 (en) * 2004-09-07 2007-12-18 The Trustees Of Boston University Methods and compositions for treating hypercholesterolemia
AR051446A1 (es) * 2004-09-23 2007-01-17 Bristol Myers Squibb Co Glucosidos de c-arilo como inhibidores selectivos de transportadores de glucosa (sglt2)
US20070134204A1 (en) * 2005-12-09 2007-06-14 Henrich Cheng Method for treating nerve injury and vector construct for the same
DK2054431T3 (da) 2006-06-09 2012-01-02 Novartis Ag Konformere af bakterielle adhæsiner
NZ593743A (en) 2008-12-04 2012-07-27 Opko Ophthalmics Llc Compositions and methods for selective inhibition of pro-angiogenic vegf isoforms
US20110319476A1 (en) 2009-02-12 2011-12-29 Opko Curna, Llc Treatment of glial cell derived neurotrophic factor (gdnf) related diseases by inhibition of natural antisense transcript to gdnf
PL2396038T3 (pl) 2009-02-12 2016-05-31 Curna Inc Leczenie chorób związanych z neurotropowym czynnikiem pochodzenia mózgowego (bdnf) poprzez zahamowanie naturalnego antysensownego transkryptu bdnf
EP2403946A4 (de) 2009-03-04 2012-11-14 Behandlung von sirtuin 1 (sirt1)-bedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts gegen sirt 1
US9464287B2 (en) 2009-03-16 2016-10-11 Curna, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (NRF2) related diseases by inhibition of natural antisense transcript to NRF2
CA2755404C (en) 2009-03-17 2020-03-24 Joseph Collard Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1
CN106237345A (zh) 2009-05-06 2016-12-21 库尔纳公司 通过针对脂质转运和代谢基因的天然反义转录物的抑制治疗脂质转运和代谢基因相关疾病
ES2609655T3 (es) 2009-05-06 2017-04-21 Curna, Inc. Tratamiento de enfermedades relacionadas con tristetraprolina (TTP) mediante inhibición de transcrito antisentido natural para TTP
JP5922017B2 (ja) 2009-05-18 2016-05-24 クルナ・インコーポレーテッド リプログラミング因子に対する天然アンチセンス転写物の抑制によるリプログラミング因子関連疾患の治療
WO2010135695A2 (en) 2009-05-22 2010-11-25 Curna, Inc. TREATMENT OF TRANSCRIPTION FACTOR E3 (TFE3) and INSULIN RECEPTOR SUBSTRATE 2 (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO TFE3
EP2435571B1 (de) 2009-05-28 2016-12-14 CuRNA, Inc. Behandlung von durch antivirale gene bedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts gegen ein antivirales gen
ES2629339T3 (es) 2009-06-16 2017-08-08 Curna, Inc. Tratamiento de enfermedades relacionadas con la paraoxonasa 1 (pon1) por inhibición de transcrito antisentido natural a pon1
KR101801404B1 (ko) 2009-06-16 2017-12-20 큐알엔에이, 인크. 콜라겐 유전자에 대한 천연 안티센스 전사체의 억제에 의한 콜라겐 유전자 관련된 질환의 치료
CA2765889A1 (en) 2009-06-24 2010-12-29 Opko Curna, Llc Treatment of tumor necrosis factor receptor 2 (tnfr2) related diseases by inhibition of natural antisense transcript to tnfr2
KR101807324B1 (ko) 2009-06-26 2017-12-08 큐알엔에이, 인크. 다운 증후군 유전자에 대한 천연 안티센스 전사체의 억제에 의한 다운 증후군 유전자 관련된 질환의 치료
CA2769665A1 (en) 2009-08-05 2011-02-10 Opko Curna, Llc Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
KR101892760B1 (ko) 2009-08-25 2018-08-28 큐알엔에이, 인크. ＩＱＧＡＰ에 대한 천연 안티센스 전사체의 억제에 의한 ＧＴＰａｓｅ 활성화 단백질을 함유하는 ＩＱ 모티프(ＩＱＧＡＰ)와 관련된 질환의 치료
ES2661813T3 (es) 2009-12-16 2018-04-04 Curna, Inc. Tratamiento de enfermedades relacionadas con la peptidasa del factor de transcripción de membrana, sitio 1 (mbtps1) mediante inhibición del transcrito antisentido natural al gen mbtps1
WO2011079261A2 (en) 2009-12-23 2011-06-30 Curna, Inc. Treatment of hepatocyte growth factor (hgf) related diseases by inhibition of natural antisense transcript to hgf
EP2515947B1 (de) 2009-12-23 2021-10-06 CuRNA, Inc. Behandlung von durch das uncoupling protein 2 (ucp2) bedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts für ucp2
JP5982288B2 (ja) 2009-12-29 2016-08-31 カッパーアールエヌエー，インコーポレイテッド 腫瘍タンパク質６３（ｐ６３）に対する天然アンチセンス転写物の阻害による腫瘍タンパク質６３関連疾患の治療
RU2615450C2 (ru) 2009-12-29 2017-04-04 Курна, Инк. Лечение заболеваний, связанных с ядерным респираторным фактором 1(nrf1), путем ингибирования природного антисмыслового транскрипта к nrf1
DK2521784T3 (en) 2010-01-04 2018-03-12 Curna Inc TREATMENT OF INTERFERON REGULATORY FACTOR 8- (IRF8) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPT TO IRF8
JP5963680B2 (ja) 2010-01-06 2016-08-03 カッパーアールエヌエー，インコーポレイテッド 膵臓発生遺伝子に対する天然アンチセンス転写物の阻害による膵臓発生遺伝子疾患の治療
WO2011085347A2 (en) 2010-01-11 2011-07-14 Opko Curna, Llc Treatment of sex hormone binding globulin (shbg) related diseases by inhibition of natural antisense transcript to shbg
ES2671877T3 (es) 2010-01-25 2018-06-11 Curna, Inc. Tratamiento de enfermedades relacionadas con la RNASA (H1) mediante inhibición del transcrito antisentido natural a RNASA H1
CN102844435B (zh) 2010-02-22 2017-05-10 库尔纳公司 通过抑制吡咯啉‑5‑羧酸还原酶1(pycr1)的天然反义转录物而治疗pycr1相关疾病
EP2556160A4 (de) 2010-04-09 2013-08-21 Curna Inc Behandlung von erkrankungen im zusammenhang mit dem fibroblastenwachstumsfaktor 21 (fgf21) mittels hemmung des natürlichen antisense-transkripts gegen fgf21
WO2011139387A1 (en) 2010-05-03 2011-11-10 Opko Curna, Llc Treatment of sirtuin (sirt) related diseases by inhibition of natural antisense transcript to a sirtuin (sirt)
TWI586356B (zh) 2010-05-14 2017-06-11 可娜公司 藉由抑制ｐａｒ４天然反股轉錄本治療ｐａｒ４相關疾病
KR20180053419A (ko) 2010-05-26 2018-05-21 큐알엔에이, 인크. 무조(無調)의 동소체 1 (atoh1)에 대한 자연 안티센스 전사체의 저해에 의한 atoh1 관련된 질환의 치료
WO2012009402A2 (en) 2010-07-14 2012-01-19 Opko Curna Llc Treatment of discs large homolog (dlg) related diseases by inhibition of natural antisense transcript to dlg
CA2813901C (en) 2010-10-06 2019-11-12 Curna, Inc. Treatment of sialidase 4 (neu4) related diseases by inhibition of natural antisense transcript to neu4
EP2630241B1 (de) 2010-10-22 2018-10-17 CuRNA, Inc. Behandlung von alpha-l-iduronidase (idua)-bedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts gegen idua
ES2677070T3 (es) 2010-10-27 2018-07-27 Curna, Inc. Tratamiento de enfermedades relacionadas con el regulador del desarrollo 1 asociado a interferón (ifrd1) mediante inhibición del transcrito antisentido natural al gen ifrd1
ES2657590T3 (es) 2010-11-23 2018-03-06 Curna, Inc. Tratamiento de enfermedades relacionadas con nanog mediante inhibición del transcrito antisentido natural a nanog
RU2620980C2 (ru) 2011-06-09 2017-05-30 Курна, Инк. Лечение заболеваний, связанных с фратаксином (fxn), путем ингибирования природного антисмыслового транскрипта fxn
US20150031750A1 (en) 2012-03-15 2015-01-29 The Scripps Research Institute Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
WO2015171918A2 (en) 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
JP6867945B2 (ja) 2014-10-03 2021-05-12 コールド スプリング ハーバー ラボラトリー 核内遺伝子出力の標的とされた増強
EP3285788A4 (de) 2015-04-23 2018-12-05 University of Massachusetts Modulation der transgenexpression eines aav-vektors
SG11201802870RA (en) 2015-10-09 2018-05-30 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
EP3390642B1 (de) 2015-12-14 2021-11-24 Cold Spring Harbor Laboratory Verfahren zur behandlung von retinitis pigmentosa 13
EP3933041B1 (de) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense-oligomere zur behandlung von autosomaldominierender geistiger behinderung
EP4104867A3 (de) 2015-12-14 2023-03-01 Cold Spring Harbor Laboratory Zusammensetzungen und verfahren zur behandlung von erkrankungen des zentralen nervensystems
CA3005245A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
WO2017106283A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of liver diseases
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
SG10202108375XA (en) 2017-08-25 2021-09-29 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
CA3105448A1 (en) 2018-07-03 2020-01-09 Elstar Therapeutics, Inc. Anti-tcr antibody molecules and uses thereof
US20210292790A1 (en) 2018-07-30 2021-09-23 Gene Therapy Research Institution Co., Ltd. Method for enhancing gene expression using aav vector
JP2023525799A (ja) 2020-05-11 2023-06-19 ストーク セラピューティクス，インク． 状態及び疾患の治療のためのｏｐａ１アンチセンスオリゴマー
CA3214757A1 (en) 2021-04-08 2022-10-13 Andreas Loew Multifuntional molecules binding to tcr and uses thereof
JP2024516168A (ja) 2021-04-22 2024-04-12 デイナ ファーバー キャンサー インスティチュート，インコーポレイテッド がんを治療するための組成物および方法

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ZA858044B (en) * 1984-11-01 1987-05-27 American Home Prod Oral vaccines
JPH04501960A (ja) * 1988-11-23 1992-04-09 ベイラー カレッジ オブ メディスン 神経細胞移植
US5082670A (en) * 1988-12-15 1992-01-21 The Regents Of The University Of California Method of grafting genetically modified cells to treat defects, disease or damage or the central nervous system
EP0448650A4 (en) * 1989-02-01 1992-05-13 The General Hospital Corporation Herpes simplex virus type i expression vector
GB8918616D0 (en) * 1989-08-15 1989-09-27 Univ Glasgow Herpes simplex virus type 1 mutant
IE903130A1 (en) * 1989-09-15 1991-03-27 Regeneron Pharma Ciliary neurotrophic factor
IE911115A1 (en) 1990-04-10 1991-10-23 Canji Inc Gene therapy for cell proliferative diseases
JPH06501160A (ja) 1990-09-17 1994-02-10 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 細胞の増殖をコントロールするための方法及び組成物
WO1992007945A1 (en) * 1990-10-30 1992-05-14 Dana Farber Cancer Institute Cell type specific alteration of levels of gene products in neural cells
CA2107789A1 (en) 1991-04-05 1992-10-06 Thomas E. Wagner Retrovirus inhibition with antisense nucleic acids complementary to packaging sequences
FR2717824B1 (fr) * 1994-03-25 1996-04-26 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
US7769626B2 (en) * 2003-08-25 2010-08-03 Tom Reynolds Determining strategies for increasing loyalty of a population to an entity

Also Published As

Publication number Publication date
ZA937051B (en) 1995-03-17
NO951121L (no) 1995-03-23
FI951404A0 (fi) 1995-03-24
HUT72987A (en) 1996-06-28
AU692423B2 (en) 1998-06-11
HU219304B (en) 2001-03-28
CA2145535A1 (en) 1994-04-14
ATE404683T1 (de) 2008-08-15
NZ256018A (en) 1997-07-27
US6756523B1 (en) 2004-06-29
NO951121D0 (no) 1995-03-23
EP0669987A1 (de) 1995-09-06
JPH08501686A (ja) 1996-02-27
AU4818093A (en) 1994-04-26
ES2312164T3 (es) 2009-02-16
CA2145535C (en) 2007-07-17
FI951404A (fi) 1995-03-24
WO1994008026A1 (en) 1994-04-14
EP0669987B1 (de) 2008-08-13
US6458529B1 (en) 2002-10-01
HU9500870D0 (en) 1995-05-29

Similar Documents

Publication Publication Date Title
DE69334238D1 (de) Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn
Sawicki et al. A composite CMV-IE enhancer/β-actin promoter is ubiquitously expressed in mouse cutaneous epithelium
GR3029913T3 (en) Treatment of cell populations.
IL188791A0 (en) Hypoxia-regulating genes
DK1000146T3 (da) 5' EST'er for non-vævsspecifikke secernerede proteiner
ATE323773T1 (de) Adenovirale vektoren, die nicht von gruppe c adenovirus typen abgeleitet sind
EP1017797A4 (de) Nicht-primaten lentivirale vektoren und verpackungssysteme
AU7891498A (en) Flower organ-specific gene and its promoter sequence
ATE307886T1 (de) Nukleotidsequenzen, proteine, medikamente und diagnoseagentien zur anwendung in krebsbehandlung
BR9813917A (pt) Vetores de expressão induzìveis hipertérmicospara terapia de genetica e métodos de uso dos mesmos
HK1028788A1 (en) Ribozymal nucleic acids cleaving ccr5 or cxcr4.
DE69331144D1 (de) Entwicklung eines vektors zur erreichung von genexpression in der epidermis transgener tiere
DE69736002D1 (de) GEN ASSOZIIERT MIT IgA GLOMERULONEPHRITIS
DE59510829D1 (de) Gentherapeutisches verfahren unter verwendung von dna-vektoren ohne selektionsmarker-gen
US10889628B2 (en) Optogenetic inhibition of overactive neuronal activity
WO1995027070A1 (de) Dna-expressionsvektoren zur verwendung in der gentherapeutischen behandlung von gefässerkrankungen
Fryer et al. Identification and characterization of a DNase hypersensitive region of the human tyrosinase gene
Hershberger et al. Genomic organization of the mouse granzyme A gene. Two mRNAs encode the same mature granzyme A with different leader peptides.
EP1887083B1 (de) Verwendung der 5'UTR des D.melanogaster ZAM Retrotransposons als "Isolator" um eine hohe Transgen-Expression zu garantieren
ATE207536T1 (de) Nukleotid sequenzen und daraus abgleitete aminosäuresequenzen vom tumorgen int 6
ATE234929T1 (de) Vektor zur expression von therapeutisch relevanten genen
Pillay Analysis of tissue-specific cytosine methylation in the CpG island of the Cas-1 gene in mouse (catalase, hydrogen peroxide)
JPH10225291A (ja) Ｍｓｓ−１様蛋白質をコードするｄｎａ
RU98123505A (ru) Толлоидподобный ген млекопитающих и кодируемый им протеин
ES2005189A6 (en) Nucleotide sequences from RESA antigen of Plasmodium falciparum

Legal Events

Date Code Title Description
8364 No opposition during term of opposition