JP2010500357A - 頭蓋内のｓｉＲＮＡの運搬を通じたハンチントンの疾患治療用の組成物、デバイス及び方法 - Google Patents

頭蓋内のｓｉＲＮＡの運搬を通じたハンチントンの疾患治療用の組成物、デバイス及び方法 Download PDF

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JP2010500357A
JP2010500357A JP2009523836A JP2009523836A JP2010500357A JP 2010500357 A JP2010500357 A JP 2010500357A JP 2009523836 A JP2009523836 A JP 2009523836A JP 2009523836 A JP2009523836 A JP 2009523836A JP 2010500357 A JP2010500357 A JP 2010500357A
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small interfering
interfering rna
disease
rna
strand
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JP2010500357A5 (enExample
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ケメラー，ウィリアム，エフ．
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Medtronic Inc
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M37/00—Other apparatus for introducing media into the body; Percutany, i.e. introducing medicines into the body by diffusion through the skin
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Medical Informatics (AREA)
  • Dermatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Anesthesiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Infusion, Injection, And Reservoir Apparatuses (AREA)
JP2009523836A 2006-08-08 2007-08-08 頭蓋内のｓｉＲＮＡの運搬を通じたハンチントンの疾患治療用の組成物、デバイス及び方法 Pending JP2010500357A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US11/501,147 US7732591B2 (en) 2003-11-25 2006-08-08 Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna
PCT/US2007/017659 WO2008021149A2 (en) 2006-08-08 2007-08-08 Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna

Publications (2)

Publication Number Publication Date
JP2010500357A true JP2010500357A (ja) 2010-01-07
JP2010500357A5 JP2010500357A5 (enExample) 2010-10-07

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JP2009523836A Pending JP2010500357A (ja) 2006-08-08 2007-08-08 頭蓋内のｓｉＲＮＡの運搬を通じたハンチントンの疾患治療用の組成物、デバイス及び方法

Country Status (6)

Country Link
US (1) US7732591B2 (enExample)
EP (1) EP2056840B1 (enExample)
JP (1) JP2010500357A (enExample)
CN (1) CN101522202B (enExample)
CA (1) CA2660153C (enExample)
WO (1) WO2008021149A2 (enExample)

Families Citing this family (12)

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US7994149B2 (en) * 2003-02-03 2011-08-09 Medtronic, Inc. Method for treatment of Huntington's disease through intracranial delivery of sirna
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US9375440B2 (en) * 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US8173614B2 (en) * 2007-01-03 2012-05-08 Medtronic, Inc. Therapeuting compositions comprising an RNAi agent and a neurotrophic factor and methods of use thereof
US8957038B2 (en) 2009-07-15 2015-02-17 Medtronic, Inc. Treatment of neurological disorders
US20170102398A1 (en) * 2014-03-05 2017-04-13 Humanetics Corporation Predicting and reducing cognitive decline based on gsk-3 levels
US10786674B2 (en) * 2016-03-08 2020-09-29 Medtronic, Inc. Medical therapy target definition
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
EP3752243A4 (en) * 2018-02-14 2021-11-17 Neuroelectrics Corporation SYSTEMS AND METHODS OF TREATING BRAIN DISEASE USING TARGETED NEUROSTIMULATION
EP4663758A2 (en) * 2018-05-09 2025-12-17 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
CA3155921A1 (en) * 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. HUNTINGTINE (HTT) TARGETING RNAI AGENT COMPOSITIONS AND METHODS OF USE THEREOF
CN113846122B (zh) * 2021-08-16 2023-08-25 中国医学科学院医学生物学研究所 一种过表达snca的腺相关病毒载体aav-snca、制备方法及其应用

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