JP2004532849A - 中枢神経系へのポリヌクレオチド薬剤の送達 - Google Patents

中枢神経系へのポリヌクレオチド薬剤の送達 Download PDF

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JP2004532849A
JP2004532849A JP2002583620A JP2002583620A JP2004532849A JP 2004532849 A JP2004532849 A JP 2004532849A JP 2002583620 A JP2002583620 A JP 2002583620A JP 2002583620 A JP2002583620 A JP 2002583620A JP 2004532849 A JP2004532849 A JP 2004532849A
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polynucleotide
agent
antisense
tissue
olfactory
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JP2004532849A5 (enExample
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クリストフ レインハード,
ウィリアム エイチ. ツー フレイ,
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カイロン コーポレイション
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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
    • C12N15/1138Non-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 receptors or cell surface proteins
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
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    • A61P35/00Antineoplastic agents
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    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
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    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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JP2002583620A 2001-04-20 2002-04-19 中枢神経系へのポリヌクレオチド薬剤の送達 Pending JP2004532849A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US28531901P 2001-04-20 2001-04-20
US28871601P 2001-05-04 2001-05-04
PCT/US2002/012527 WO2002086105A1 (en) 2001-04-20 2002-04-19 Delivery of polynucleotide agents to the central nervous sysstem

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JP2008282272A Division JP2009067805A (ja) 2001-04-20 2008-10-31 中枢神経系へのポリヌクレオチド薬剤の送達

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JP2004532849A true JP2004532849A (ja) 2004-10-28
JP2004532849A5 JP2004532849A5 (enExample) 2005-12-22

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JP2002583620A Pending JP2004532849A (ja) 2001-04-20 2002-04-19 中枢神経系へのポリヌクレオチド薬剤の送達
JP2008282272A Withdrawn JP2009067805A (ja) 2001-04-20 2008-10-31 中枢神経系へのポリヌクレオチド薬剤の送達

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US (3) US20030165434A1 (enExample)
EP (1) EP1385937A4 (enExample)
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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008545394A (ja) * 2005-05-18 2008-12-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 生存細胞中でのリアルタイム核酸分析のための組成物、方法およびキット
JP2015510889A (ja) * 2012-03-09 2015-04-13 ノースイースタン・ユニバーシティ 中枢神経系障害を治療するための核酸ナノ粒子を中枢神経系へ送達する方法
WO2023243606A1 (ja) * 2022-06-13 2023-12-21 国立大学法人 東京大学 核酸とカチオン性ポリマーとのポリイオンコンプレックスであって、正の表面電位を有し、核酸を脳組織に送達することができるポリイオンコンプレックス

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1423537A4 (en) * 2001-08-07 2006-11-29 Univ Delaware COMPOSITIONS AND METHODS FOR PREVENTING AND TREATING HUNTINGTON'S DISEASE
US20040096880A1 (en) * 2001-08-07 2004-05-20 Kmiec Eric B. Compositions and methods for the treatment of diseases exhibiting protein misassembly and aggregation
CA2515484C (en) 2003-02-11 2011-09-20 Antisense Therapeutics Ltd Modulation of insulin like growth factor i receptor expression
US20050271651A1 (en) * 2004-01-23 2005-12-08 Oklahoma Medical Research Foundation Inhibition of bright function as a treatment for excessive immunoglobulin production
US9216161B2 (en) 2004-08-13 2015-12-22 Healthpartners Research Foundation Methods of treating Huntington's disease comprising administering metal chelators to the upper one-third of the nasal cavity
US7776312B2 (en) * 2004-08-13 2010-08-17 Healthpartners Research Foundation Method of treating Alzheimer's disease comprising administering deferoxamine (DFO) to the upper one-third of the nasal cavity
US7618615B2 (en) 2004-08-13 2009-11-17 Healthpartners Research Foundation Methods for providing neuroprotection for the animal central nervous system against neurodegeneration caused by ischemia
CN101048139A (zh) * 2004-10-28 2007-10-03 艾德克斯实验室公司 药物活性化合物的控释组合物
US20060165739A1 (en) * 2005-01-06 2006-07-27 Mary Kay Inc. Alcohol-free microemulsion composition
US20070054843A1 (en) 2005-08-26 2007-03-08 Yeomans David C Methods for treatment of headaches by administration of oxytocin
US7754679B2 (en) * 2005-11-16 2010-07-13 Idexx Laboratories, Inc. Pharmaceutical compositions for the administration of aptamers
CA2626368A1 (en) * 2005-11-16 2007-05-24 Idexx Laboratories Inc. Pharmaceutical compositions for the administration of aptamers
US8114440B2 (en) * 2005-11-16 2012-02-14 Idexx Laboratories Inc. Pharmaceutical compositions for the administration of aptamers
US20080261893A1 (en) * 2006-02-24 2008-10-23 Denise Barbut Topical corneal analgesia using neurotensin receptor agonists and synergistic neurotensin combinations without delaying wound healing
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
WO2007127163A2 (en) 2006-04-24 2007-11-08 Geron Corporation Cns-tumor treatment method and composition
AU2008225100A1 (en) * 2007-03-12 2008-09-18 Mayo Foundation For Medical Education And Research Topical corneal analgesia using neurotensin receptor agonists and synergistic neurotensin combinations without delaying wound healing
US9707274B2 (en) 2007-06-08 2017-07-18 Healthpartners Research & Education Methods for preventing and treating post-traumatic stress disorder (PTSD)
US8828960B2 (en) * 2007-07-17 2014-09-09 Idexx Laboratories, Inc. Amino acid vitamin ester compositions for controlled delivery of pharmaceutically active compounds
AU2009271149A1 (en) * 2008-07-14 2010-01-21 Oklahoma Medical Research Foundation Production of pluripotent cells through inhibition of Bright/ARID3a function
EP2862929B1 (en) 2009-12-09 2017-09-06 Quark Pharmaceuticals, Inc. Compositions and methods for treating diseases, disorders or injury of the CNS
US9821159B2 (en) 2010-11-16 2017-11-21 The Board Of Trustees Of The Leland Stanford Junior University Stimulation devices and methods
JP2013542838A (ja) 2010-11-16 2013-11-28 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー ドライアイを治療するためのシステムおよび方法
US8609088B2 (en) 2011-05-10 2013-12-17 Regents Of The University Of Minnesota Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
US8987199B2 (en) 2011-06-15 2015-03-24 Nerve Access, Inc. Pharmaceutical compositions for intranasal administration for the treatment of neurodegenerative disorders
WO2013016223A2 (en) * 2011-07-22 2013-01-31 The University Of Chicago Treatments for migraine and related disorders
US10279012B2 (en) 2013-03-11 2019-05-07 Healthpartners Research & Education Methods of treating and preventing social communication disorder in patients by intranasal administration of insulin
EP2967817B1 (en) 2013-03-12 2021-03-10 Oculeve, Inc. Implant delivery devices and systems
HK1220934A1 (zh) 2013-04-19 2017-05-19 Oculeve, Inc. 鼻刺激装置和方法
EP3622821A1 (en) 2013-05-15 2020-03-18 Regents of the University of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
EP3689338A1 (en) 2014-02-25 2020-08-05 Oculeve, Inc. Polymer formulations for nasolacrimal stimulation
US10314911B2 (en) 2014-04-08 2019-06-11 Healthpartners Research & Education Methods for protecting and treating traumatic brain injury, concussion and brain inflammation with intranasal insulin
DK3171928T3 (da) 2014-07-25 2020-05-18 Oculeve Inc Stimulationsmønstre til behandling af tørt øje
AU2015335774B2 (en) 2014-10-22 2020-07-16 Oculeve, Inc. Implantable nasal stimulator systems and methods
WO2016065215A1 (en) 2014-10-22 2016-04-28 Oculeve, Inc. Stimulation devices and methods for treating dry eye
AU2015335772B2 (en) 2014-10-22 2020-07-09 Oculeve, Inc. Contact lens for increasing tear production
HRP20231438T1 (hr) 2015-01-07 2024-06-07 Tonix Pharma Limited Formulacije oksitocina koje sadrže magnezij i načini uporabe
US10335435B2 (en) 2015-05-22 2019-07-02 Marco Merida Method for endoscopically delivering stem cells to the brain using an intranasal, injectable approach
US10426958B2 (en) 2015-12-04 2019-10-01 Oculeve, Inc. Intranasal stimulation for enhanced release of ocular mucins and other tear proteins
US10252048B2 (en) 2016-02-19 2019-04-09 Oculeve, Inc. Nasal stimulation for rhinitis, nasal congestion, and ocular allergies
CN115814055A (zh) 2016-04-12 2023-03-21 三叉神经股份公司 含镁催产素制剂和应用方法
IL262211B2 (en) 2016-04-15 2024-01-01 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
WO2017192572A1 (en) 2016-05-02 2017-11-09 Oculeve, Inc. Intranasal stimulation for treatment of meibomian gland disease and blepharitis
CA3045018A1 (en) 2016-12-02 2018-06-07 Oculeve, Inc. Apparatus and method for dry eye forecast and treatment recommendation
CN109498832B (zh) * 2017-09-15 2021-10-08 中国药科大学 一种交联多孔止血微球及其制备方法
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
AU2020346186A1 (en) 2019-09-09 2022-04-07 Seurat Therapeutics Combination therapy for the treatment of migraines
WO2021076883A2 (en) * 2019-10-16 2021-04-22 Brown University Muscle regeneration and growth

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5624898A (en) * 1989-12-05 1997-04-29 Ramsey Foundation Method for administering neurologic agents to the brain
US5714170A (en) * 1994-11-16 1998-02-03 Thomas Jefferson University Method of inducing resistance to tumor growth
US6025193A (en) * 1996-03-15 2000-02-15 Allegheny University Of The Health Sciences Methods and compositions for diagnosis and treatment of pathological conditions related to abnormal dopamine receptor expression
US5814500A (en) * 1996-10-31 1998-09-29 The Johns Hopkins University School Of Medicine Delivery construct for antisense nucleic acids and methods of use
IL131212A0 (en) * 1999-08-03 2001-01-28 Yissum Res Dev Co Recombinant virus and live-virus vaccines

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008545394A (ja) * 2005-05-18 2008-12-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 生存細胞中でのリアルタイム核酸分析のための組成物、方法およびキット
JP2015510889A (ja) * 2012-03-09 2015-04-13 ノースイースタン・ユニバーシティ 中枢神経系障害を治療するための核酸ナノ粒子を中枢神経系へ送達する方法
WO2023243606A1 (ja) * 2022-06-13 2023-12-21 国立大学法人 東京大学 核酸とカチオン性ポリマーとのポリイオンコンプレックスであって、正の表面電位を有し、核酸を脳組織に送達することができるポリイオンコンプレックス

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EP1385937A4 (en) 2005-11-09
WO2002086105A1 (en) 2002-10-31
EP1385937A1 (en) 2004-02-04
US20030165434A1 (en) 2003-09-04
US20090264506A1 (en) 2009-10-22
JP2009067805A (ja) 2009-04-02

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