JP7027328B2 - タウ発現を減少させるための組成物および方法 - Google Patents

タウ発現を減少させるための組成物および方法 Download PDF

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JP7027328B2
JP7027328B2 JP2018550880A JP2018550880A JP7027328B2 JP 7027328 B2 JP7027328 B2 JP 7027328B2 JP 2018550880 A JP2018550880 A JP 2018550880A JP 2018550880 A JP2018550880 A JP 2018550880A JP 7027328 B2 JP7027328 B2 JP 7027328B2
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oligonucleotide
tau
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mapt
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JP2019500428A (ja
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オフェンガイム，マニュエラ ポリドーロ
ヴァイラー，ヤン
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ノバルティス アーゲー
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JP2018550880A 2015-12-21 2016-12-19 タウ発現を減少させるための組成物および方法 Active JP7027328B2 (ja)

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JP2021076622A JP2021118732A (ja) 2015-12-21 2021-04-28 タウ発現を減少させるための組成物および方法
JP2022021494A JP7594557B2 (ja) 2015-12-21 2022-02-15 タウ発現を減少させるための組成物および方法
JP2024203529A JP2025026976A (ja) 2015-12-21 2024-11-21 タウ発現を減少させるための組成物および方法

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US201562270165P 2015-12-21 2015-12-21
US62/270,165 2015-12-21
PCT/IB2016/057794 WO2017109679A1 (en) 2015-12-21 2016-12-19 Compositions and methods for decreasing tau expression

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Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013203395A1 (en) 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
TWI772856B (zh) 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
ES2963428T3 (es) 2016-09-29 2024-03-27 Biogen Ma Inc Compuestos y métodos para reducir la expresión de Tau
KR20200131263A (ko) 2018-03-13 2020-11-23 얀센 파마슈티카 엔.브이. 변형 올리고뉴클레오티드 및 타우병증에서의 사용 방법
SG11202012759XA (en) 2018-07-03 2021-01-28 Hoffmann La Roche Oligonucleotides for modulating tau expression
AU2021246024A1 (en) * 2020-03-30 2022-10-27 Alnylam Pharmaceuticals, Inc. Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof
CN113817728A (zh) * 2020-06-19 2021-12-21 四川大学华西医院 一种有效干扰Tau蛋白表达的重组慢病毒及其应用
EP4355878A4 (en) * 2021-06-16 2025-04-09 Empirico Inc. TREATMENT OF MTRES1-RELATED DISEASES AND DISORDERS
WO2023288111A2 (en) * 2021-07-16 2023-01-19 Aptah Bio, Inc. Polynucleotide compositions and methods for gene expression regulation
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
WO2023102188A1 (en) * 2021-12-03 2023-06-08 Quralis Corporation Gapmer antisense oligonucleotides with modified backbone chemistries
WO2024091874A1 (en) * 2022-10-25 2024-05-02 Empirico Inc. Gene therapy treatment of mtres1 related diseases and disorders
EP4652278A1 (en) * 2023-01-18 2025-11-26 Aptah Bio, Inc. Polynucleotide compositions and methods for treatment of cancer
JP2026504808A (ja) * 2023-01-18 2026-02-10 アプター バイオ，インク． 神経変性疾患の処置のためのポリヌクレオチド組成物および方法
WO2024220930A2 (en) * 2023-04-20 2024-10-24 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
WO2025188990A1 (en) * 2024-03-08 2025-09-12 Eli Lilly And Company Mapt antisense oligonucleotide
US12551569B2 (en) 2024-03-31 2026-02-17 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use
WO2026025082A1 (en) * 2024-07-25 2026-01-29 Biogen Ma Inc. Methods for treating alzheimer's disease

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020010947A1 (en) 2000-01-21 2002-01-24 Gurney Mark E. Transgenic mouse model of human neurodegenerative disease
WO2015010135A2 (en) 2013-07-19 2015-01-22 Isis Pharmaceuticals, Inc. Compositions for modulating tau expression
JP2015516953A (ja) 2012-03-30 2015-06-18 ワシントン・ユニバーシティＷａｓｈｉｎｇｔｏｎ Ｕｎｉｖｅｒｓｉｔｙ 発作を減少させるためおよび神経変性症候群を改質するためにタウ発現を調節する方法

Family Cites Families (103)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3817837A (en) 1971-05-14 1974-06-18 Syva Corp Enzyme amplification assay
US3939350A (en) 1974-04-29 1976-02-17 Board Of Trustees Of The Leland Stanford Junior University Fluorescent immunoassay employing total reflection for activation
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4275149A (en) 1978-11-24 1981-06-23 Syva Company Macromolecular environment control in specific receptor assays
US4277437A (en) 1978-04-05 1981-07-07 Syva Company Kit for carrying out chemically induced fluorescence immunoassay
US4366241A (en) 1980-08-07 1982-12-28 Syva Company Concentrating zone method in heterogeneous immunoassays
AU651569B2 (en) 1990-01-11 1994-07-28 Isis Pharmaceuticals, Inc. Compositions and methods for detecting and modulating RNA activity and gene expression
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US20020052331A1 (en) 1990-08-14 2002-05-02 Brenda Baker Compositions and methods for antisense inhibition of protein translation
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
ATE398176T1 (de) 1991-12-06 2008-07-15 Max Planck Gesellschaft Phosphorylierungsepitope aus dem tau-protein zur diagnose und behandlung der alzheimer-krankheit
US7408027B1 (en) 1991-12-06 2008-08-05 Max-Planck-Gesellschaft Zur Forderung Der Wissenchaften Tools for the diagnosis and treatment of Alzheimer's disease
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
EP0670897A4 (en) * 1992-10-05 1997-08-06 Isis Pharmaceuticals Inc INHIBITION OF THE ras GENE USING NON-CODING OLIGONUCLEOTIDES.
KR940021073A (ko) 1993-03-02 1994-10-17 미야베 요시가즈 알쯔하이머병의 예방 또는 치료제, 그 스크리닝 방법 및 인간 유래의 타우 단백질 키나아제 i
CA2116460A1 (en) 1993-03-02 1994-09-03 Mitsubishi Chemical Corporation Preventive or therapeutic agents for alzheimer's disease, a screening method of alzheimer's disease and tau-protein kinase i originated from human being
CN1123038A (zh) 1993-05-11 1996-05-22 北卡罗来纳大学查珀尔希尔分校 阻止异常剪接的反义寡核苷酸及其使用方法
ATE267207T1 (de) 1996-02-14 2004-06-15 Isis Pharmaceuticals Inc Kohlenhydratmodifizierte luckenhafte oligonukleotide
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US20040186071A1 (en) 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
US6664443B1 (en) 1998-05-01 2003-12-16 Mayo Foundation For Medical Education And Research Pathogenic Tau mutations in transgenic mice
CA2330944A1 (en) 1998-05-01 1999-11-11 Mayo Foundation For Medical Education And Research Pathogeniv tau mutations
CN100419079C (zh) * 1998-07-14 2008-09-17 科里克萨有限公司 用于治疗和诊断前列腺癌的组合物和方法
CZ296576B6 (cs) 1999-02-12 2006-04-12 Sankyo Company Limited Nukleosidový analog a oligonukleotidový analog a farmaceutický prostredek, sonda a primer s jeho obsahem
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US7098192B2 (en) * 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
US6350868B1 (en) 1999-04-26 2002-02-26 University Of North Carolina At Chapel Hill Antisense human fucosyltransferase sequences and methods of use thereof
EP1178999B1 (en) 1999-05-04 2007-03-14 Santaris Pharma A/S L-ribo-lna analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
CN101284873A (zh) * 1999-06-30 2008-10-15 科里克萨有限公司 用于肺癌治疗和诊断的组合物和方法
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
KR20030016233A (ko) * 2000-02-28 2003-02-26 디코드 제네틱스 이에이치에프 사람 정신분열증 유전자
AU2002231741A1 (en) 2001-01-11 2002-07-24 Bayer Aktiengesellschaft Regulation of human tau-tubulin kinase
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
US20050130924A1 (en) 2002-06-26 2005-06-16 Monia Brett P. Antisense inhibition via RNAse H-independent reduction in mRNA
AU2003247521A1 (en) 2002-06-26 2004-01-19 Isis Pharmaceuticals, Inc. Antisense inhibition via rnase h-independent reduction in mrna
US20050106731A1 (en) 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US20080176812A1 (en) 2002-08-05 2008-07-24 Davidson Beverly L Allele-specific silencing of disease genes
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
ES2352668T3 (es) 2002-08-14 2011-02-22 Mitsubishi Chemical Medience Corporation Anticuerpo específico para una proteína tau del sistema nervioso central.
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US8163896B1 (en) 2002-11-14 2012-04-24 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7250496B2 (en) * 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7906326B2 (en) 2003-05-07 2011-03-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides associated with alzheimer's disease and uses thereof
US7790867B2 (en) 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
US7696334B1 (en) 2002-12-05 2010-04-13 Rosetta Genomics, Ltd. Bioinformatically detectable human herpesvirus 5 regulatory gene
US20130130231A1 (en) 2002-11-26 2013-05-23 Isaac Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US20070077553A1 (en) 2003-10-30 2007-04-05 Rosetta Genomics Bioinformatically detectable group of novel vaccinia regulatory genes and uses thereof
US20060257851A1 (en) 2002-11-26 2006-11-16 Itzhak Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
AU2003904328A0 (en) 2003-08-13 2003-08-28 Garvan Institute Of Medical Research Diagnosis and treatment of neurodegenerative disorders
US7888497B2 (en) 2003-08-13 2011-02-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
CN1845993B (zh) 2003-08-28 2010-06-23 诺瓦提斯公司 具有平端和3＇修饰的干扰rna双链体
JPWO2005030959A1 (ja) * 2003-09-25 2006-12-07 千葉県 神経芽細胞腫予後診断のためのマイクロアレイと神経芽細胞腫予後診断方法
WO2005040382A2 (de) 2003-10-28 2005-05-06 Alexander Cherkasky Cherkasky - fusionsproteine enthaltend antikorperbinde-, antigenbinde - mikrotubulibinde und immunantwortauslosende
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US7687616B1 (en) 2004-05-14 2010-03-30 Rosetta Genomics Ltd Small molecules modulating activity of micro RNA oligonucleotides and micro RNA targets and uses thereof
CA2566519C (en) * 2004-05-14 2020-04-21 Rosetta Genomics Ltd. Micrornas and uses thereof
ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
US7312035B2 (en) 2004-09-03 2007-12-25 Affymetrix, Inc. Methods of genetic analysis of yeast
US7833513B2 (en) 2004-12-03 2010-11-16 Rhode Island Hospital Treatment of Alzheimer's Disease
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
CN101102789B (zh) 2004-12-03 2012-05-30 罗得岛医院 阿尔茨海默病的诊断和治疗
US20070048756A1 (en) 2005-04-18 2007-03-01 Affymetrix, Inc. Methods for whole genome association studies
WO2007028065A2 (en) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
US7901882B2 (en) 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
CA2648132C (en) 2006-04-03 2019-05-28 Santaris Pharma A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
US20090280110A1 (en) 2006-06-27 2009-11-12 University Of Virginia Patent Foundation Cell Model for Alzheimer's Disease Pathology
US20100021914A1 (en) 2006-11-23 2010-01-28 Querdenker Aps Oligonucleotides for modulating target rna activity
WO2008124066A1 (en) 2007-04-05 2008-10-16 The J. David Gladstone Institutes Agents that reduce neuronal overexcitation
JP2008307007A (ja) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
EP2268811A1 (en) 2008-03-07 2011-01-05 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
ES2599979T3 (es) 2009-04-24 2017-02-06 Roche Innovation Center Copenhagen A/S Composiciones farmacéuticas para el tratamiento de pacientes de VHC que no responden al interferón
LT2499249T (lt) 2009-11-12 2018-12-27 The University Of Western Australia Priešprasmės molekulės ir patologijų gydymo būdai
US20150018223A1 (en) 2010-01-28 2015-01-15 University Of Massachusetts Lowell Methods of diagnosing tau-associated neurodegenerative diseases
US20130079505A1 (en) 2010-03-24 2013-03-28 Mirrx Therapeutics A/S Bivalent antisense oligonucleotides
EP3260540A1 (en) 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
US9145557B2 (en) 2010-11-23 2015-09-29 Mirrx Therapeutics A/S Oligonucleotides for modulation of target RNA activity
DK2655621T3 (en) 2010-12-20 2018-08-13 Massachusetts Gen Hospital Polycomb-associated non-coding RNAS
US9957558B2 (en) * 2011-04-28 2018-05-01 Life Technologies Corporation Methods and compositions for multiplex PCR
WO2013013165A2 (en) 2011-07-21 2013-01-24 The Trustees Of The University Of Pennsylvania Use of lna compounds for therapeutic inhibition of mir-21 in systemic lupus erythematosus
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
EP2855520B1 (en) 2012-06-04 2018-09-26 Novartis AG Site-specific labeling methods and molecules produced thereby
CN104936977B (zh) 2012-10-24 2019-10-08 开普敦大学 微管修饰化合物
US9200068B2 (en) 2012-12-18 2015-12-01 Regents Of The University Of Minnesota Compositions and methods related to tauopathy
CA2894899A1 (en) 2012-12-20 2014-06-26 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
NZ731587A (en) 2013-03-14 2021-07-30 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
RS58529B1 (sr) 2013-03-14 2019-04-30 Sarepta Therapeutics Inc Sastavi koji preskaču egzon za tretiranje mišićne distrofije
ES2807379T3 (es) 2013-03-14 2021-02-22 Ionis Pharmaceuticals Inc Composiciones y métodos para regular la expresión de Tau
CA2951816A1 (en) 2013-06-12 2014-12-18 Oncoimmunin, Inc. Systemic in vivo delivery of oligonucleotides
WO2015068075A2 (en) 2013-10-24 2015-05-14 Institut National De La Sante Et De La Recherche Medicale Detection of tau
CN104711324A (zh) * 2013-12-11 2015-06-17 芮屈生物技术（上海）有限公司 老年性痴呆病变前期TAU基因的mRNA水平原位杂交筛查试剂盒
EP3736334A1 (en) * 2014-07-16 2020-11-11 Arrowhead Pharmaceuticals, Inc. Rnai compositions to treat apoc3-related diseases
WO2016019063A1 (en) 2014-07-29 2016-02-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
EP3254104B1 (en) 2015-02-04 2021-08-25 Bristol-Myers Squibb Company Methods of selecting therapeutic molecules
WO2016127002A1 (en) 2015-02-04 2016-08-11 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
AU2016215155A1 (en) 2015-02-04 2017-08-17 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
US20170035860A1 (en) * 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020010947A1 (en) 2000-01-21 2002-01-24 Gurney Mark E. Transgenic mouse model of human neurodegenerative disease
JP2015516953A (ja) 2012-03-30 2015-06-18 ワシントン・ユニバーシティＷａｓｈｉｎｇｔｏｎ Ｕｎｉｖｅｒｓｉｔｙ 発作を減少させるためおよび神経変性症候群を改質するためにタウ発現を調節する方法
WO2015010135A2 (en) 2013-07-19 2015-01-22 Isis Pharmaceuticals, Inc. Compositions for modulating tau expression

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