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

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/185Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44Oxidoreductases (1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0006Oxidoreductases (1.) acting on CH-OH groups as donors (1.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y114/00Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16Oxidoreductases 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)
    • C12Y114/16002Tyrosine 3-monooxygenase (1.14.16.2)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01051Alpha-L-fucosidase (3.2.1.51)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • 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)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Psychology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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) US6458529B1 (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) FI951404A0 (de)
HU (1) HU219304B (de)
NO (1) NO951121L (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
CA2108144A1 (en) 1991-03-06 1992-09-07 Jack A. Roth 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
US6410010B1 (en) 1992-10-13 2002-06-25 Board Of Regents, The University Of Texas System Recombinant P53 adenovirus compositions
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
US6080569A (en) 1993-06-24 2000-06-27 Merck & Co., Inc. Adenovirus vectors generated from helper viruses and helper-dependent vectors
US6730507B1 (en) 1993-06-24 2004-05-04 Merck & Co., Inc. Use of helper-dependent adenoviral vectors of alternative serotypes permits repeat vector administration
US6140087A (en) * 1993-06-24 2000-10-31 Advec, Inc. Adenovirus vectors for gene therapy
EP0705344B8 (de) * 1993-06-24 2006-05-10 Advec Inc. Adenovirus vektoren für gentherapie
US7045347B2 (en) 1993-06-24 2006-05-16 Advec, Inc. Helper dependent adenovirus vectors based on integrase family site-specific recombinases
US20020136708A1 (en) 1993-06-24 2002-09-26 Graham Frank L. System for production of helper dependent adenovirus vectors based on use of endonucleases
US6120764A (en) * 1993-06-24 2000-09-19 Advec, Inc. Adenoviruses for control of gene expression
FR2716460B1 (fr) * 1994-02-21 2002-08-09 Rhone Poulenc Rorer Sa Modèle animal de la maladie d'Alzheimer, préparation et utilisations.
FR2717497B1 (fr) * 1994-03-18 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
FR2717495B1 (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.
CA2189067A1 (en) * 1994-04-28 1995-11-09 Gary J. Nabel Gene delivery vector using plasmid dna packaged into an adenovirus and a packaging cell line
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
PT787200E (pt) * 1994-10-28 2005-08-31 Univ Pennsylvania Adenovirus melhorado e metodos para a sua utilizacao
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
US6127525A (en) * 1995-02-21 2000-10-03 Cornell Research Foundation, Inc. Chimeric adenoviral coat protein and methods of using same
US5770442A (en) * 1995-02-21 1998-06-23 Cornell Research Foundation, Inc. Chimeric adenoviral fiber protein and methods of using same
US6974694B2 (en) 1995-06-07 2005-12-13 Advec, Inc. Adenoviruses for control of gene expression
US6783980B2 (en) 1995-06-15 2004-08-31 Crucell Holland B.V. Packaging systems for human recombinant adenovirus to be used in gene therapy
JP4051416B2 (ja) 1995-06-15 2008-02-27 クルーセル ホランド ベスローテン フェンノートシャップ 遺伝子治療に使用されるヒト組換えアデノウイルス用のパッケージングシステム
WO1997017090A1 (en) * 1995-11-07 1997-05-15 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
CA2671261A1 (en) 1997-11-06 1999-05-20 Novartis Vaccines And Diagnostics S.R.L. Neisserial antigens
AU2144999A (en) * 1998-01-13 1999-08-02 Claude Gravel Viral vectors encoding neurofilament heavy proteins and their use
WO1999036559A1 (en) * 1998-01-13 1999-07-22 Julien Jean Pierre Viral vectors expressing self-polymerizing neuronal intermediate filaments and their use
WO1999036560A2 (en) * 1998-01-13 1999-07-22 Julien Jean Pierre Viral vectors encoding neurofilament light proteins and their use
ATE446368T1 (de) 1998-01-14 2009-11-15 Novartis Vaccines & Diagnostic Antigene aus neisseria meningitidis
EP2261355A3 (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
DK1228217T3 (da) 1999-04-30 2013-02-25 Novartis Vaccines & Diagnostic Konserverede neisseria-antigener
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
CA2864069A1 (en) 1999-10-29 2001-05-03 Novartis Vaccines And Diagnostics S.R.L. Neisserial antigenic peptides
ES2507100T3 (es) 2000-01-17 2014-10-14 Novartis Vaccines And Diagnostics S.R.L. Vacuna OMV suplementada contra meningococo
AU2001253233B2 (en) * 2000-04-06 2006-12-07 Kos Pharmaceuticals, Inc. Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia
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
EP2189473A3 (de) 2000-10-27 2010-08-11 Novartis Vaccines and Diagnostics S.r.l. Nukleinsäure und Proteine von Gruppen A und B Streptokokken
GB0107661D0 (en) 2001-03-27 2001-05-16 Chiron Spa Staphylococcus aureus
GB0107658D0 (en) 2001-03-27 2001-05-16 Chiron Spa Streptococcus pneumoniae
FR2825372B1 (fr) * 2001-06-01 2004-06-18 Centre Nat Rech Scient Pseudotypage des vecteurs vih-1 par des enveloppes de rhabdovirus
JP4413617B2 (ja) 2001-12-12 2010-02-10 カイロン ソチエタ ア レスポンサビリタ リミタータ Chlamydiatrachomatisに対する免疫化
US20030158112A1 (en) 2002-02-15 2003-08-21 Johns Hopkins University School Of Medicine Selective induction of apoptosis to treat ocular disease
MXPA04010282A (es) 2002-04-18 2005-08-18 Acuity Pharmaceuticals Inc Medios y metodos para la inhibicion especifica de genes en celulas y tejido de cns y/u ojo.
ES2335657T3 (es) 2002-04-25 2010-03-31 Crucell Holland B.V. Medios y metodos para la produccion de vectores de adenovirus.
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
WO2005012538A2 (en) 2003-08-01 2005-02-10 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Accelerated vaccination
US8268324B2 (en) 2004-03-29 2012-09-18 Galpharma Co., Ltd. Modified galectin 9 proteins and use thereof
EP1796712A4 (de) * 2004-09-07 2009-11-11 Kos Life Sciences Inc Verfahren und zusammensetzungen zur behandlung von hypercholesterolämie
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
PL2054431T3 (pl) 2006-06-09 2012-07-31 Novartis Ag Konformery adhezyn bakteryjnych
US8470792B2 (en) 2008-12-04 2013-06-25 Opko Pharmaceuticals, Llc. Compositions and methods for selective inhibition of VEGF
EP2396038B1 (de) 2009-02-12 2015-10-21 CuRNA, Inc. Behandlung von erkrankungen im zusammenhang mit dem brain-derived neurotrophen faktor (bdnf) mittels hemmung des natürlichen antisense-transkripts gegen bdnf
ES2658626T3 (es) 2009-02-12 2018-03-12 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado de células gliales (GDNF) por inhibición de transcrito antisentido natural a GDNF
EP2963116B1 (de) 2009-03-04 2020-11-11 CuRNA, Inc. Behandlung von sirtuin 1 (sirt1)-bedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts gegen sirt 1
ES2656290T3 (es) 2009-03-16 2018-02-26 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor nuclear (derivado de eritroide 2) similar al 2 (NRF2) mediante inhibición del transcrito antisentido natural a NRF2
WO2010107740A2 (en) 2009-03-17 2010-09-23 Curna, Inc. Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1
US20120046236A1 (en) 2009-05-06 2012-02-23 Opko Curna Llc Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp
US9155754B2 (en) 2009-05-06 2015-10-13 Curna, Inc. Treatment of ABCA1 gene related diseases by inhibition of a natural antisense transcript to ABCA1
KR101749356B1 (ko) 2009-05-18 2017-07-06 큐알엔에이, 인크. 재편성 인자에 대한 천연 안티센스 전사체의 억제에 의한 재편성 인자 관련된 질환의 치료
EP2432882B1 (de) 2009-05-22 2019-12-25 CuRNA, Inc. Behandlung von erkrankungen im zusammenhang mit dem transkriptionsfaktor e3 (tfe3) und dem insulinrezeptorsubstrat 2 (irs2) mittels hemmung des natürlichen antisense-transkripts gegen tfe3
WO2010138806A2 (en) 2009-05-28 2010-12-02 Curna, Inc. Treatment of antiviral gene related diseases by inhibition of natural antisense transcript to an antiviral gene
CA2765700C (en) 2009-06-16 2021-01-05 Opko Curna, Llc Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
EP2443238B1 (de) 2009-06-16 2017-03-22 CuRNA, Inc. Behandlung von erkrankungen im zusammenhang mit paraoxonase 1 (pon1) mittels hemmung des natürlichen antisense-transkripts gegen pon 1
JP6073133B2 (ja) 2009-06-24 2017-02-01 クルナ・インコーポレーテッド 腫瘍壊死因子受容体2(tnfr2)に対する天然アンチセンス転写物の抑制によるtnfr2関連疾患の治療
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
JP6128848B2 (ja) 2009-08-05 2017-05-17 クルナ・インコーポレーテッド インスリン遺伝子(ins)に対する天然アンチセンス転写物の抑制によるインスリン遺伝子(ins)関連疾患の治療
WO2011031482A2 (en) 2009-08-25 2011-03-17 Curna, Inc. Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap
US9173895B2 (en) 2009-12-16 2015-11-03 Curna, Inc. Treatment of membrane bound transcription factor peptidase, site 1 (MBTPS1) related diseases by inhibition of natural antisense transcript to MBTPS1
CA2782375C (en) 2009-12-23 2023-10-31 Opko Curna, Llc Treatment of uncoupling protein 2 (ucp2) related diseases by inhibition of natural antisense transcript to ucp2
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
JP5993744B2 (ja) 2009-12-29 2016-09-14 カッパーアールエヌエー,インコーポレイテッド 核呼吸因子1(nrf1)に対する天然アンチセンス転写物の阻害による核呼吸因子1関連疾患の治療
WO2011090741A2 (en) 2009-12-29 2011-07-28 Opko Curna, Llc TREATMENT OF TUMOR PROTEIN 63 (p63) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO p63
US8946181B2 (en) 2010-01-04 2015-02-03 Curna, Inc. Treatment of interferon regulatory factor 8 (IRF8) related diseases by inhibition of natural antisense transcript to IRF8
US8912157B2 (en) 2010-01-06 2014-12-16 Curna, Inc. Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to a pancreatic developmental gene
RU2611191C2 (ru) 2010-01-11 2017-02-21 Курна, Инк. Лечение заболеваний, связанных со связывающим половые гормоны глобулином (гспг), путем ингибирования природного антисмыслового транскрипта к гспг
JP5981850B2 (ja) 2010-01-25 2016-08-31 カッパーアールエヌエー,インコーポレイテッド RNaseH1に対する天然アンチセンス転写物の阻害によるRNaseH1関連疾患の治療
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
TWI644675B (zh) 2010-04-09 2018-12-21 可娜公司 藉由抑制纖維母細胞生長因子21(fgf21)之天然反義轉錄物以治療fgf21相關疾病
KR101992076B1 (ko) 2010-05-03 2019-06-21 큐알엔에이, 인크. 시르투인 (sirt)에 대한 자연 안티센스 전사체의 저해에 의한 시르투인 (sirt) 관련된 질환의 치료
TWI531370B (zh) 2010-05-14 2016-05-01 可娜公司 藉由抑制par4天然反股轉錄本治療par4相關疾病
EP3299464B1 (de) 2010-05-26 2019-10-02 CuRNA, Inc. Behandlung von durch atonal homolog 1 (atoh1) vermittelten erkrankungen durch hemmung des natürlichen antisense-transkripts für atoh1
NO2593547T3 (de) 2010-07-14 2018-04-14
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
US9222088B2 (en) 2010-10-22 2015-12-29 Curna, Inc. Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
US20130210893A1 (en) 2010-10-27 2013-08-15 Curna, Inc. Treatment of interferon-related developmental regulator 1 (ifrd1) related diseases by inhibition of natural antisense transcript to ifrd1
JP6071893B2 (ja) 2010-11-23 2017-02-01 カッパーアールエヌエー,インコーポレイテッド Nanogへの天然アンチセンス転写物の阻害によるnanog関連疾患の治療
US9593330B2 (en) 2011-06-09 2017-03-14 Curna, Inc. Treatment of frataxin (FXN) related diseases by inhibition of natural antisense transcript to FXN
WO2013138374A2 (en) 2012-03-15 2013-09-19 Curna, Inc. 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
SG11201702682PA (en) 2014-10-03 2017-04-27 Cold Spring Harbor Lab Targeted augmentation of nuclear gene output
WO2016172155A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
CA3000971A1 (en) 2015-10-09 2017-04-13 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
CA3005245A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
CA3005090A1 (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
EP3389725B1 (de) 2015-12-14 2022-04-06 Cold Spring Harbor Laboratory Zusammensetzungen und verfahren zur behandlung von erkrankungen des zentralen nervensystems
JP7049248B2 (ja) 2015-12-14 2022-04-06 コールド スプリング ハーバー ラボラトリー 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー
JP7054675B2 (ja) 2015-12-14 2022-04-14 コールド スプリング ハーバー ラボラトリー 網膜色素変性症18と網膜色素変性症13の処置のための組成物と方法
AU2018322319B2 (en) 2017-08-25 2021-08-05 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
EP3818083A2 (de) 2018-07-03 2021-05-12 Elstar Therapeutics, Inc. Anti-tcr-antikörpermoleküle und ihre verwendungen
WO2020026968A1 (ja) 2018-07-30 2020-02-06 株式会社遺伝子治療研究所 Aavベクターによる遺伝子発現を増強する方法
US20230287410A1 (en) 2020-05-11 2023-09-14 Stoke Therapeutics, Inc. Opa1 antisense oligomers for treatment of conditions and diseases
KR20240004462A (ko) 2021-04-08 2024-01-11 마렝고 테라퓨틱스, 인크. Tcr에 결합하는 다기능성 분자 및 이의 용도
CA3214540A1 (en) 2021-04-22 2022-10-27 Pasi A. Janne Compositions and methods for treating cancer

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
AU4669189A (en) * 1988-11-23 1990-06-12 Baylor College Of Medicine Neural cell transplantation
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
WO1990009441A1 (en) * 1989-02-01 1990-08-23 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
IE20010427A1 (en) 1990-04-10 2003-03-05 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
AU655279B2 (en) 1991-04-05 1994-12-15 Edison Animal Biotechnology Center 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

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