CA2464887A1 - Methods and compositions for treating parkinson's disease - Google Patents

Methods and compositions for treating parkinson's disease Download PDF

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Publication number
CA2464887A1
CA2464887A1 CA002464887A CA2464887A CA2464887A1 CA 2464887 A1 CA2464887 A1 CA 2464887A1 CA 002464887 A CA002464887 A CA 002464887A CA 2464887 A CA2464887 A CA 2464887A CA 2464887 A1 CA2464887 A1 CA 2464887A1
Authority
CA
Canada
Prior art keywords
aav
cell
cells
nurrl
expression
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.)
Abandoned
Application number
CA002464887A
Other languages
English (en)
French (fr)
Inventor
Stephen Fawell
Orla Conneely
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.)
Baylor College of Medicine
Biogen MA Inc
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA2464887A1 publication Critical patent/CA2464887A1/en
Abandoned 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
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1783Nuclear receptors, e.g. retinoic acid receptor [RAR], RXR, nuclear orphan receptors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/18Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/24Antidepressants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Psychiatry (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • High Energy & Nuclear Physics (AREA)
  • Pain & Pain Management (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
CA002464887A 2001-10-30 2002-10-30 Methods and compositions for treating parkinson's disease Abandoned CA2464887A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US34100901P 2001-10-30 2001-10-30
US60/341,009 2001-10-30
PCT/US2002/034613 WO2003037260A2 (en) 2001-10-30 2002-10-30 Methods and compositions for treating parkinson's disease

Publications (1)

Publication Number Publication Date
CA2464887A1 true CA2464887A1 (en) 2003-05-08

Family

ID=23335891

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002464887A Abandoned CA2464887A1 (en) 2001-10-30 2002-10-30 Methods and compositions for treating parkinson's disease

Country Status (5)

Country Link
US (1) US20050070493A1 (https=)
EP (1) EP1469730A4 (https=)
JP (1) JP2005507927A (https=)
CA (1) CA2464887A1 (https=)
WO (1) WO2003037260A2 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005075990A2 (en) * 2004-02-07 2005-08-18 Bayer Healthcare Ag Diagnostics and therapeutics for diseases associated with human nuclear receptor nr4a2 (nr4a2)
US20120301463A1 (en) 2009-09-30 2012-11-29 President And Fellows Of Harvard College Methods for Modulation of Autophagy Through the Modulation of Autophagy-Enhancing Gene Products
EP3235908A1 (en) 2016-04-21 2017-10-25 Ecole Normale Superieure De Lyon Methods for selectively modulating the activity of distinct subtypes of cells
CN112955174A (zh) 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
US20220008557A1 (en) * 2018-11-14 2022-01-13 Flagship Pioneering Innovations V, Inc. Fusosome compositions for cns delivery
CN113631718A (zh) 2018-11-14 2021-11-09 旗舰先锋创新V股份有限公司 用于特定隔室货物递送的组合物和方法
US20220226269A1 (en) * 2019-06-12 2022-07-21 President And Fellows Of Harvard College Methods and compositions for modulation of an interspecies gut bacterial pathway for levodopa metabolism
KR20240028975A (ko) 2021-04-08 2024-03-05 사나 바이오테크놀로지, 인크. Cd8-특이적 항체 구조체들 및 이의 조성물

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683195A (en) * 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5173414A (en) * 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5869337A (en) * 1993-02-12 1999-02-09 President And Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US5830462A (en) * 1993-02-12 1998-11-03 President & Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US5589362A (en) * 1993-06-14 1996-12-31 Basf Aktiengesellschaft Tetracycline regulated transcriptional modulators with altered DNA binding specificities
US5464758A (en) * 1993-06-14 1995-11-07 Gossen; Manfred Tight control of gene expression in eucaryotic cells by tetracycline-responsive promoters
JPH09500526A (ja) * 1993-06-14 1997-01-21 ベーアーエスエフ アクツィエンゲゼルシャフト テトラサイクリン反応性プロモーターによる真核細胞の遺伝子発現の厳密な制御
ATE386131T1 (de) * 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
WO1999061066A2 (en) * 1998-05-27 1999-12-02 Avigen, Inc. Convection-enhanced delivery of aav vectors
US6284539B1 (en) * 1998-10-09 2001-09-04 Neuralstem Biopharmaceuticals, Ltd. Method for generating dopaminergic cells derived from neural precursors
US6312949B1 (en) * 1999-03-26 2001-11-06 The Salk Institute For Biological Studies Regulation of tyrosine hydroxylase expression
DE60035191T2 (de) * 1999-05-03 2008-06-19 Neuro Therapeutics Ab Materialien und methoden zur entwicklung von dopaminergen neuronen
WO2003012040A2 (en) * 2001-07-27 2003-02-13 Baylor College Of Medecine Mutant nurr1 gene in parkinson's disease

Also Published As

Publication number Publication date
EP1469730A4 (en) 2006-02-01
JP2005507927A (ja) 2005-03-24
WO2003037260A2 (en) 2003-05-08
EP1469730A2 (en) 2004-10-27
WO2003037260A3 (en) 2004-08-19
US20050070493A1 (en) 2005-03-31

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