CN106030302B - 治疗神经变性疾病 - Google Patents

治疗神经变性疾病 Download PDF

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Publication number
CN106030302B
CN106030302B CN201480075396.4A CN201480075396A CN106030302B CN 106030302 B CN106030302 B CN 106030302B CN 201480075396 A CN201480075396 A CN 201480075396A CN 106030302 B CN106030302 B CN 106030302B
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laccer
b4galt6
astrocytes
progressive
eae
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CN106030302A (zh
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F·J·昆塔纳
L·梅奥
H·韦纳
R·哈尔斯
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The Brigham and Women's Hospital Inc
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The Brigham and Women's Hospital Inc
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    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/564—Immunoassay; Biospecific binding assay; Materials therefor for pre-existing immune complex or autoimmune disease, i.e. systemic lupus erythematosus, rheumatoid arthritis, multiple sclerosis, rheumatoid factors or complement components C1-C9
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715—Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/716—Glucans
    • 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
    • A—HUMAN NECESSITIES
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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
    • C12N15/1137—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 against enzymes
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
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    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2400/00—Assays, e.g. immunoassays or enzyme assays, involving carbohydrates
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    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2405/00—Assays, e.g. immunoassays or enzyme assays, involving lipids
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/285—Demyelinating diseases; Multipel sclerosis
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
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    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Rehabilitation Therapy (AREA)
  • General Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Food Science & Technology (AREA)
  • Pathology (AREA)
  • Cell Biology (AREA)
  • Rheumatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Neurosurgery (AREA)
CN201480075396.4A 2013-12-12 2014-12-12 治疗神经变性疾病 Active CN106030302B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361963738P 2013-12-12 2013-12-12
US61/963,738 2013-12-12
US201462049813P 2014-09-12 2014-09-12
US62/049,813 2014-09-12
PCT/US2014/070099 WO2015089443A2 (en) 2013-12-12 2014-12-12 Treating neurodegenerative disease

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CN106030302A CN106030302A (zh) 2016-10-12
CN106030302B true CN106030302B (zh) 2019-09-24

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US (1) US9927437B2 (enExample)
EP (1) EP3080602B1 (enExample)
JP (1) JP6550388B2 (enExample)
KR (1) KR20160089528A (enExample)
CN (1) CN106030302B (enExample)
CA (1) CA2933554A1 (enExample)
WO (1) WO2015089443A2 (enExample)

Families Citing this family (8)

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US10512656B2 (en) 2016-04-21 2019-12-24 Baylor College Of Medicine Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction
JP7084689B2 (ja) * 2016-12-08 2022-06-15 公益財団法人東京都医学総合研究所 脳組織の異常を伴う非ヒト動物の作製方法およびその利用
WO2019030645A1 (en) * 2017-08-08 2019-02-14 Kashiv Pharma Llc Pharmaceutical composition comprising eliglustat
JP2021515037A (ja) 2018-02-26 2021-06-17 アントルクス，インコーポレーテッド 寛容原性リポソーム及びその使用方法
WO2019169317A1 (en) * 2018-03-02 2019-09-06 Stueve Olaf Methods and compositions for treating natalizumab-associated progressive multifocal encephalopathy
EP3833398A4 (en) * 2018-09-21 2021-12-22 University of Connecticut COMPOSITIONS AND METHODS OF RESTORING PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN'S SYNDROME IN HUMANS
CN114717232A (zh) * 2020-12-22 2022-07-08 未来智人再生医学研究院(广州)有限公司 表达irf-1靶向抑制因子的多能干细胞及其衍生物与应用
CN116794308B (zh) * 2023-07-20 2026-05-26 中国药科大学 β-1,4-半乳糖基转移酶1及其抑制剂在制备治疗急慢性肝病药物中的应用

Citations (5)

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FR2869915A1 (fr) * 2004-05-07 2005-11-11 Merck Sante Soc Par Actions Si Gm3 synthase, comme cible therapeutique dans les complications microvasculaires du diabete
CN102388307A (zh) * 2008-11-12 2012-03-21 耶达研究与发展有限公司 多发性硬化的诊断
CN102994554A (zh) * 2011-09-15 2013-03-27 花王株式会社 神经酰胺和/或葡萄糖神经酰胺产生促进剂的制造方法
CN103059160A (zh) * 2011-10-20 2013-04-24 中国科学院上海药物研究所 β-葡聚糖GFPBW1及其制备方法和用途
EP2640385A1 (en) * 2010-11-18 2013-09-25 Synta Pharmaceuticals Corp. Preselection of subjects for therapeutic treatment based on hypoxic status

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US5700909A (en) * 1993-07-30 1997-12-23 The Regents Of The University Of California Prosaposin and cytokine-derived peptides
EP1825853A2 (en) * 1998-07-27 2007-08-29 Johns Hopkins University Methods for treating conditions modulated by lactosyceramide
US20070270350A1 (en) * 2003-12-23 2007-11-22 Musc Foundation For Research Development Methods and Compositions for the Prevention and Treatment of Inflammatory Diseases or Conditions
WO2005123055A2 (en) * 2004-06-14 2005-12-29 Musc Foundation For Research Development Methods for treating inflammatory disorders
US7572592B2 (en) * 2005-01-31 2009-08-11 Glycominds Ltd Method for diagnosing multiple sclerosis
US8263576B2 (en) * 2006-08-15 2012-09-11 Mayo Foundation For Medical Education And Research Non-natural sphingolipid analogs and uses thereof
AU2011349256B2 (en) * 2010-12-20 2017-01-05 Scidec Therapeutics, Inc. Methods for treating neurological conditions and compositions and materials therefor

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2869915A1 (fr) * 2004-05-07 2005-11-11 Merck Sante Soc Par Actions Si Gm3 synthase, comme cible therapeutique dans les complications microvasculaires du diabete
CN102388307A (zh) * 2008-11-12 2012-03-21 耶达研究与发展有限公司 多发性硬化的诊断
EP2640385A1 (en) * 2010-11-18 2013-09-25 Synta Pharmaceuticals Corp. Preselection of subjects for therapeutic treatment based on hypoxic status
CN102994554A (zh) * 2011-09-15 2013-03-27 花王株式会社 神经酰胺和/或葡萄糖神经酰胺产生促进剂的制造方法
CN103059160A (zh) * 2011-10-20 2013-04-24 中国科学院上海药物研究所 β-葡聚糖GFPBW1及其制备方法和用途

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US20160313324A1 (en) 2016-10-27
KR20160089528A (ko) 2016-07-27
JP6550388B2 (ja) 2019-07-24
EP3080602A4 (en) 2017-07-05
WO2015089443A3 (en) 2015-11-12
US9927437B2 (en) 2018-03-27
EP3080602A2 (en) 2016-10-19
CA2933554A1 (en) 2015-06-18
EP3080602B1 (en) 2018-11-28
JP2017502278A (ja) 2017-01-19
WO2015089443A2 (en) 2015-06-18
CN106030302A (zh) 2016-10-12

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