JP2013529181A - 神経障害を治療するための組成物および方法 - Google Patents

神経障害を治療するための組成物および方法 Download PDF

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JP2013529181A
JP2013529181A JP2013501487A JP2013501487A JP2013529181A JP 2013529181 A JP2013529181 A JP 2013529181A JP 2013501487 A JP2013501487 A JP 2013501487A JP 2013501487 A JP2013501487 A JP 2013501487A JP 2013529181 A JP2013529181 A JP 2013529181A
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pkd1
antibody
gene
nucleic acid
cells
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JP2013529181A5 (enExample
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エム. フィンクバイナー,スティーブン
ラームナート ラオ,ヴィクラム
ローズマリー カーター,サラ
スザンヌ レドー,エバ
ジュ キム,ホン
カンピオーニ,マシュー
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ザ ジェイ. デヴィッド グラッドストーン インスティテューツ
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Publication of JP2013529181A publication Critical patent/JP2013529181A/ja
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/506Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular 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
    • 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
    • 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
    • A61P25/28Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/40Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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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    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/70Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77Internalization into the cell
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2013501487A 2010-03-25 2011-03-24 神経障害を治療するための組成物および方法 Pending JP2013529181A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US31759810P 2010-03-25 2010-03-25
US61/317,598 2010-03-25
PCT/US2011/029806 WO2011119842A1 (en) 2010-03-25 2011-03-24 Compositions and methods for treating neurological disorders

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JP2013529181A true JP2013529181A (ja) 2013-07-18
JP2013529181A5 JP2013529181A5 (enExample) 2014-05-08

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US (2) US9359445B2 (enExample)
EP (1) EP2550361B1 (enExample)
JP (1) JP2013529181A (enExample)
WO (1) WO2011119842A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018151223A1 (ja) * 2017-02-16 2018-08-23 国立大学法人京都大学 細胞評価方法、細胞評価装置、及び細胞評価プログラム
JP2020510909A (ja) * 2017-02-09 2020-04-09 コグノア,インク. デジタル個別化医療のためのプラットフォームとシステム

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160178610A1 (en) * 2013-08-07 2016-06-23 Friedrich Miescher Institute For Biomedical Research New screening method for the treatment Friedreich's ataxia
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
CA3070446A1 (en) 2017-07-25 2019-01-31 Immutics, Inc. Treating cancer by blocking the interaction of tim-3 and its ligand
KR20240035631A (ko) 2017-08-25 2024-03-15 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
WO2019213525A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
CN120058944A (zh) 2019-01-30 2025-05-30 真和制药有限公司 抗gal3抗体及其用途
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머
EP4157338A4 (en) 2020-05-26 2024-11-13 TrueBinding, Inc. METHOD FOR TREATING INFLAMMATORY DISEASES BY GALECTIN-3 BLOCKING

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009147246A1 (en) * 2008-06-06 2009-12-10 Medizinische Universität Graz Compounds reducing or inhibiting the expression of pkd1 for diagnosis and therapy of brain tumors

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー.ファウ. 修飾リボザイム
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
CA2154363A1 (en) 1993-01-22 1994-08-04 Bruce A. Sullenger Localization of therapeutic agents
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US7964598B2 (en) 1995-10-17 2011-06-21 The J. David Gladstone Institutes ApoE4 domain interaction inhibitors and methods of use thereof
DE69637256T2 (de) 1996-01-16 2008-06-19 Sirna Therapeutics, Inc., Boulder Synthese von Methoxynukleoside und enzymatische Nukleisäure Moleküle
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
DE19650758C1 (de) * 1996-12-06 1998-01-02 Deutsches Krebsforsch PKD1-Fragmente mit Bindungsregionen für PKD1-spezifische Antikörper
US6124272A (en) * 1999-04-09 2000-09-26 Isis Pharmaceutical Inc. Antisense modulation of PDK-1 expression
US20050233329A1 (en) 2002-02-20 2005-10-20 Sirna Therapeutics, Inc. Inhibition of gene expression using duplex forming oligonucleotides
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
CA2526831C (en) 2001-05-18 2012-07-31 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (sina)
GB0117326D0 (en) 2001-07-16 2001-09-05 Univ Aberdeen Napthoquinone-type inhibitors of protein aggregation
AR036604A1 (es) 2001-09-21 2004-09-22 Mitsubishi Pharma Corp Derivados de 4-pirimidona 3-sustituida
US20040121407A1 (en) * 2002-09-06 2004-06-24 Elixir Pharmaceuticals, Inc. Regulation of the growth hormone/IGF-1 axis
JP2007511618A (ja) 2003-11-19 2007-05-10 シグナル ファーマシューティカルズ,エルエルシー インダゾール化合物およびタンパク質キナーゼ阻害剤としてのその使用方法
PT1893612E (pt) 2005-06-22 2011-11-21 Plexxikon Inc Derivados de pirrolo [2,3-b]piridina como inibidores de proteína quinase
GB0602178D0 (en) * 2006-02-03 2006-03-15 Merck Sharp & Dohme Therapeutic treatment
AU2007245496A1 (en) 2006-04-26 2007-11-08 Cancer Research Technology Limited Amino-ethyl-amino-aryl (AEAA) compounds and their use
US20090175852A1 (en) 2006-06-06 2009-07-09 Schering Corporation Imidazopyrazines as protein kinase inhibitors
US8240498B2 (en) 2006-10-31 2012-08-14 Crown Packaging Technology, Inc. Resealable closure
US8618063B2 (en) * 2008-04-09 2013-12-31 The Board Of Trustees Of The University Of Illinois Method for treating a synucleinopathy
ES2385276B1 (es) 2010-02-25 2013-07-05 Universidad Del País Vasco Compuestos para el tratamiento de alzheimer.

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009147246A1 (en) * 2008-06-06 2009-12-10 Medizinische Universität Graz Compounds reducing or inhibiting the expression of pkd1 for diagnosis and therapy of brain tumors

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JPN6015012230; Abstract of the Annual Meeting of the Society for Neuroscience Vol.39, 200811, 334.5/D72 *
JPN6015012231; Abstract of the Annual Meeting of the Society for Neuroscience Vol.39, 2009, 137.7/C58 *

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020510909A (ja) * 2017-02-09 2020-04-09 コグノア,インク. デジタル個別化医療のためのプラットフォームとシステム
JP7324709B2 (ja) 2017-02-09 2023-08-10 コグノア,インク. デジタル個別化医療のためのプラットフォームとシステム
WO2018151223A1 (ja) * 2017-02-16 2018-08-23 国立大学法人京都大学 細胞評価方法、細胞評価装置、及び細胞評価プログラム
JPWO2018151223A1 (ja) * 2017-02-16 2019-12-26 国立大学法人京都大学 細胞評価方法、細胞評価装置、及び細胞評価プログラム
US11436719B2 (en) 2017-02-16 2022-09-06 Kyoto University Cell evaluation method, cell evaluation device, and recording medium storing cell evaluation program
US12511736B2 (en) 2017-02-16 2025-12-30 Kyoto University Cell evaluation method, cell evaluation device, and cell evaluation program

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EP2550361A4 (en) 2014-04-30
EP2550361A1 (en) 2013-01-30
EP2550361B1 (en) 2017-02-08
US20130095113A1 (en) 2013-04-18
US9359445B2 (en) 2016-06-07
WO2011119842A1 (en) 2011-09-29
US20160354372A1 (en) 2016-12-08

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