JP2018521014A5 - - Google Patents

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Publication number
JP2018521014A5
JP2018521014A5 JP2017561811A JP2017561811A JP2018521014A5 JP 2018521014 A5 JP2018521014 A5 JP 2018521014A5 JP 2017561811 A JP2017561811 A JP 2017561811A JP 2017561811 A JP2017561811 A JP 2017561811A JP 2018521014 A5 JP2018521014 A5 JP 2018521014A5
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JP
Japan
Prior art keywords
composition
amyloidosis
disease
group
trim
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JP2017561811A
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English (en)
Japanese (ja)
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JP2018521014A (ja
JP7011467B2 (ja
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Priority claimed from PCT/US2016/034751 external-priority patent/WO2016196328A1/en
Publication of JP2018521014A publication Critical patent/JP2018521014A/ja
Publication of JP2018521014A5 publication Critical patent/JP2018521014A5/ja
Priority to JP2021155978A priority Critical patent/JP2022000459A/ja
Application granted granted Critical
Publication of JP7011467B2 publication Critical patent/JP7011467B2/ja
Priority to JP2024135688A priority patent/JP2024161501A/ja
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Anticipated expiration legal-status Critical

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JP2017561811A 2015-05-29 2016-05-27 ミスフォールドタンパク質の分解のための組成物及び方法 Active JP7011467B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021155978A JP2022000459A (ja) 2015-05-29 2021-09-24 ミスフォールドタンパク質の分解のための組成物及び方法
JP2024135688A JP2024161501A (ja) 2015-05-29 2024-08-15 ミスフォールドタンパク質の分解のための組成物及び方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562168309P 2015-05-29 2015-05-29
US62/168,309 2015-05-29
PCT/US2016/034751 WO2016196328A1 (en) 2015-05-29 2016-05-27 Compositions and methods for degradation of misfolded proteins

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021155978A Division JP2022000459A (ja) 2015-05-29 2021-09-24 ミスフォールドタンパク質の分解のための組成物及び方法

Publications (3)

Publication Number Publication Date
JP2018521014A JP2018521014A (ja) 2018-08-02
JP2018521014A5 true JP2018521014A5 (https=) 2019-06-20
JP7011467B2 JP7011467B2 (ja) 2022-02-10

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ID=57441753

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2017561811A Active JP7011467B2 (ja) 2015-05-29 2016-05-27 ミスフォールドタンパク質の分解のための組成物及び方法
JP2021155978A Pending JP2022000459A (ja) 2015-05-29 2021-09-24 ミスフォールドタンパク質の分解のための組成物及び方法
JP2024135688A Pending JP2024161501A (ja) 2015-05-29 2024-08-15 ミスフォールドタンパク質の分解のための組成物及び方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2021155978A Pending JP2022000459A (ja) 2015-05-29 2021-09-24 ミスフォールドタンパク質の分解のための組成物及び方法
JP2024135688A Pending JP2024161501A (ja) 2015-05-29 2024-08-15 ミスフォールドタンパク質の分解のための組成物及び方法

Country Status (15)

Country Link
US (2) US11542500B2 (https=)
EP (1) EP3302706A4 (https=)
JP (3) JP7011467B2 (https=)
KR (2) KR20240108531A (https=)
CN (1) CN108271364A (https=)
AU (3) AU2016271635B2 (https=)
BR (1) BR112017025574A2 (https=)
CA (1) CA2987057A1 (https=)
HK (1) HK1252757A1 (https=)
IL (3) IL255912B2 (https=)
MX (3) MX2017015373A (https=)
RU (2) RU2021134920A (https=)
SG (1) SG10202011833TA (https=)
WO (1) WO2016196328A1 (https=)
ZA (1) ZA201708067B (https=)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
CN110753557B (zh) * 2017-03-22 2024-03-22 神济昌华(北京)生物科技有限公司 Trim72通过泛素化fus蛋白突变体作为als的潜在治疗性靶标
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN109609614B (zh) * 2017-09-30 2022-07-15 首都医科大学附属北京胸科医院 检测trim2、trim4、trim32和/或trim46基因或蛋白产品的用途
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
CN108018303A (zh) * 2017-12-20 2018-05-11 江苏浩欧博生物医药股份有限公司 一种人Ro52重组蛋白的制备方法
WO2019175881A1 (en) 2018-03-14 2019-09-19 Technion Research & Development Foundation Limited A prognostic biomarker in cancer
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
US11147815B2 (en) 2018-08-21 2021-10-19 Synkrino Biotherapeutics, Inc. Compositions and methods for treating diseases associated with uncontrolled inflammatory responses
EP3863642A4 (en) * 2018-10-09 2022-06-29 The Regents of the University of California Covalent targeting of e3 ligases
WO2020227515A1 (en) * 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
CN110302383A (zh) * 2019-07-29 2019-10-08 深圳先进技术研究院 Trim11基因和蛋白靶点以及抑制剂在乳腺癌中的用途
CN110669791B (zh) * 2019-10-29 2020-08-04 成都益安博生物技术有限公司 一种提高细胞抗体表达量的方法
KR102114880B1 (ko) * 2020-01-15 2020-05-26 서울대학교병원 코돈 최적화된 TIF1γ 폴리뉴클레오티드를 포함하는 재조합 벡터 및 이의 용도
GB202005876D0 (en) * 2020-04-22 2020-06-03 Univ Dundee Protein degradation
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CN113292658B (zh) * 2021-04-17 2023-02-28 复旦大学 一种融合蛋白及其在靶向降解细胞内蛋白中的应用
CN113651879A (zh) * 2021-08-18 2021-11-16 武汉华美生物工程有限公司 一种trim21全长蛋白的制备方法及应用
CN117159747B (zh) * 2023-08-30 2024-12-20 暨南大学 一种亨廷顿舞蹈症的药物及其制备方法
CN118272444A (zh) * 2024-02-29 2024-07-02 宜昌市中心人民医院(三峡大学第一临床医学院、三峡大学附属中心人民医院) 一种慢病毒载体LV-Trim69及其在制备改善心脏衰老药物上的应用
CN120209082B (zh) * 2025-05-28 2025-08-05 中南大学 一种靶向阻断trim25与brd7蛋白结合的多肽及其应用

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20010027182A1 (en) * 1998-05-29 2001-10-04 Huda Y. Zoghbi Chaperone suppression of ataxin-1 aggregation and altered subcellular proteasome localization imply protein misfilind in sca1
WO2001090342A2 (en) 2000-05-22 2001-11-29 Johns Hopkins University Genetic engineering of vascular grafts to resist disease
US20040235733A1 (en) * 2003-02-27 2004-11-25 Steffan Joan S. Methods and reagents for reducing polyglutamine toxicity
US20050215562A1 (en) * 2003-06-23 2005-09-29 Patrick Tremblay Methods for treating protein aggregation disorders
WO2005081911A2 (en) * 2004-02-25 2005-09-09 Dana-Farber Cancer Institute, Inc. METHODS AND COMPOSITIONS FOR THE TREATMENT AND PREVENTION OF HIV INFECTION USING TRIM5α
US20140056873A1 (en) 2004-06-10 2014-02-27 University Of Leicester Methods for Treating Conditions Associated with MASP-2 Dependent Complement Activation
WO2006116716A2 (en) * 2005-04-27 2006-11-02 University Of Florida Materials and methods for enhanced degradation of mutant proteins associated with human disease
EP1870107A1 (en) * 2006-06-23 2007-12-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Use of inducer of promyelocytic leukemia protein (PML) for treating polyglutamine expansion neurodegenerative diseases
US7785857B2 (en) 2006-08-31 2010-08-31 Saint Louis University Protein C variant
CA2749163A1 (en) 2009-01-14 2010-07-22 The Salk Institute For Biological Studies Methods for screening and compounds that protect against amyloid diseases
EP2826860B1 (en) * 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
GB201012410D0 (en) * 2010-07-23 2010-09-08 Medical Res Council Intracellular immunity
AU2011295629B2 (en) 2010-09-01 2014-05-01 Organ Transport Pty Ltd Perfusion composition
CN103127492A (zh) * 2011-11-23 2013-06-05 中国科学院上海巴斯德研究所 一种抗丙型肝炎病毒的蛋白及其用途
TR201905612T4 (tr) * 2012-12-05 2019-05-21 Univ Heidelberg Ruprecht Karls Proteinlerin ve çok değerlikli, hücreye nüfuz eden peptitlerin konjugatları ve bunların kullanımları.
US20160136123A1 (en) * 2013-06-14 2016-05-19 Vojo P. Deretic Treatment of autophagy-related disorders
WO2016054554A1 (en) * 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs

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