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

治疗神经变性疾病 Download PDF

Info

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
Authority
CN
China
Prior art keywords
laccer
b4galt6
astrocytes
inhibitor
eae
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.)
Active
Application number
CN201480075396.4A
Other languages
English (en)
Chinese (zh)
Other versions
CN106030302A (zh
Inventor
F·J·昆塔纳
L·梅奥
H·韦纳
R·哈尔斯
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.)
Brigham and Womens Hospital Inc
Original Assignee
Brigham and Womens Hospital Inc
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 Brigham and Womens Hospital Inc filed Critical Brigham and Womens Hospital Inc
Publication of CN106030302A publication Critical patent/CN106030302A/zh
Application granted granted Critical
Publication of CN106030302B publication Critical patent/CN106030302B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • 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
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • 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
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • 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
    • A61K31/33—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
    • 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
    • A61K31/7088—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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/11—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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2400/00—Assays, e.g. immunoassays or enzyme assays, involving carbohydrates
    • G—PHYSICS
    • 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
    • G01N2800/50—Determining the risk of developing a disease
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Rheumatology (AREA)
  • Pathology (AREA)
  • Rehabilitation Therapy (AREA)
  • Food Science & Technology (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • General Physics & Mathematics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Neurology (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

Publications (2)

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

Family

ID=53371971

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201480075396.4A Active CN106030302B (zh) 2013-12-12 2014-12-12 治疗神经变性疾病

Country Status (7)

Country Link
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 (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20200197366A1 (en) * 2017-08-08 2020-06-25 Kashiv Biosciences, 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
AU2019344006B2 (en) 2018-09-21 2025-11-27 University Of Connecticut Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome
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

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
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

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP2007516294A (ja) * 2003-12-23 2007-06-21 エムユーエスシー ファウンデーション フォー リサーチ ディベロップメント 炎症性の疾患または症状の予防および治療のための方法および組成物
US20090111812A1 (en) * 2004-06-14 2009-04-30 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
WO2012088133A1 (en) * 2010-12-20 2012-06-28 Stephen Davies 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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Monoclonal Antibody Specific for Lactosylceramide;Frank W等;《THE JOURNAL OF BIOLOGICACLH EMISTRY》;19830915;6008-6012 *

Also Published As

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

Similar Documents

Publication Publication Date Title
CN106030302B (zh) 治疗神经变性疾病
Kwon et al. SGK1 inhibition in glia ameliorates pathologies and symptoms in Parkinson disease animal models
Gentile et al. Siponimod (BAF312) prevents synaptic neurodegeneration in experimental multiple sclerosis
Mayo et al. Regulation of astrocyte activation by glycolipids drives chronic CNS inflammation
Bhattacharya et al. Proteomics implicates peptidyl arginine deiminase 2 and optic nerve citrullination in glaucoma pathogenesis
US9248128B2 (en) Method for enhancing remyelination using GLI1 inhibitors
Smith et al. The impact of regional astrocyte interferon-γ signaling during chronic autoimmunity: a novel role for the immunoproteasome
Cacabelos et al. Neuroimmune crosstalk in CNS disorders: the histamine connection
Jensen et al. Targeting TNFα produced by astrocytes expressing amyotrophic lateral sclerosis‐linked mutant fused in sarcoma prevents neurodegeneration and motor dysfunction in mice
Clarkson et al. ISGylation is induced in neurons by demyelination driving ISG15-dependent microglial activation
Liu et al. Atsttrin reduces lipopolysaccharide-induced neuroinflammation by inhibiting the nuclear factor kappa B signaling pathway
Mayo et al. B4GALT6 regulates astrocyte activation during CNS inflammation
WO2017180841A1 (en) Treatment of cerebral cavernous malformations
CN110121361A (zh) 用于治疗运动神经元疾病的神经节苷脂代谢抑制剂
US20250152711A1 (en) Compositions and methods for modulating the immune system
US20250051458A1 (en) Methods for Treating Neurodegeneration Targeting IGF1/IGF1R
US20240301408A1 (en) Microrna 195 compositions and methods for treating cognitive impairment
WO2021127008A1 (en) Compositions and methods for treating neuromuscular disorders
HK1230277A1 (en) Treating neurodegenerative disease
HK1230277B (en) Treating neurodegenerative disease
US20250090568A1 (en) Method of treating long-covid induced neurologic diseases
Mora et al. Juxtacrine DLL4-NOTCH1 signaling between astrocytes drives neuroinflammation via the IL-6-STAT3 axis
Bai et al. miR‐365a‐3p Alleviates Isoflurane‐Induced Neuroinflammation and Cognitive Impairment by Targeting MyD88 to Inhibit NF‐κB Signaling
WO2023102215A1 (en) Compositions and methods for treating pain
US20240009274A1 (en) Use of NRG-1Beta1 for Detection and/or Treatment of Multiple Sclerosis

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant