JP2022543722A - 機能的ニューロン死に関連する神経系疾患の予防および／または治療におけるＰｔｂｐ１阻害剤の使用 - Google Patents

機能的ニューロン死に関連する神経系疾患の予防および／または治療におけるＰｔｂｐ１阻害剤の使用 Download PDF

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JP2022543722A
JP2022543722A JP2021554990A JP2021554990A JP2022543722A JP 2022543722 A JP2022543722 A JP 2022543722A JP 2021554990 A JP2021554990 A JP 2021554990A JP 2021554990 A JP2021554990 A JP 2021554990A JP 2022543722 A JP2022543722 A JP 2022543722A
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ptbp1
cells
gene
aav
neurons
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JPWO2021032068A5 (https=
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フイ ヤン
ハイボ ゾウ
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Center for Excellence in Brain Science and Intelligence Technology Chinese Academy of Sciences
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Center for Excellence in Brain Science and Intelligence Technology Chinese Academy of Sciences
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    • A—HUMAN NECESSITIES
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
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  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021554990A 2019-08-16 2020-08-17 機能的ニューロン死に関連する神経系疾患の予防および／または治療におけるＰｔｂｐ１阻害剤の使用 Withdrawn JP2022543722A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
CN201910760367 2019-08-16
CN201910760367.6 2019-08-16
CN201911046435.9 2019-10-30
CN201911046435 2019-10-30
PCT/CN2020/081489 WO2021031565A1 (en) 2019-08-16 2020-03-26 Treatment of Neuronal Diseases
CNPCT/CN2020/081489 2020-03-26
CN202010740568.2 2020-07-28
CN202010740568.2A CN112451669A (zh) 2019-08-16 2020-07-28 Ptbp1抑制剂在预防和/或治疗功能性神经元死亡相关的神经系统疾病中的应用
PCT/CN2020/109653 WO2021032068A1 (zh) 2019-08-16 2020-08-17 Ptbp1抑制剂在预防和/或治疗功能性神经元死亡相关的神经系统疾病中的应用

Publications (3)

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JP2022543722A true JP2022543722A (ja) 2022-10-14
JPWO2021032068A5 JPWO2021032068A5 (https=) 2023-08-01
JP2022543722A5 JP2022543722A5 (https=) 2023-08-01

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US (2) US20220273726A1 (https=)
EP (1) EP4039801A4 (https=)
JP (1) JP2022543722A (https=)
WO (2) WO2021032068A1 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023150553A1 (en) * 2022-02-01 2023-08-10 University Of Rochester Gpr17 promoter-based targeting and transduction of glial progenitor cells
WO2022246204A2 (en) * 2021-05-21 2022-11-24 Ionis Pharmaceuticals, Inc. Compounds for reducing ptbp1 expression
US20250161495A1 (en) * 2022-02-01 2025-05-22 University Of Rochester Methods of generating a population of neurons from human glial progenitor cells and genetic constructs for carrying out such methods

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
PT1916258E (pt) 1999-08-09 2014-07-29 Genzyme Corp Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
KR20090071603A (ko) * 2006-09-19 2009-07-01 노파르티스 아게 Ｒａｆ 억제제에 대한 표적 조절, 효능, 진단 및/또는 예후의 바이오마커
EP2104734A2 (en) * 2006-12-08 2009-09-30 Asuragen, INC. Mir-20 regulated genes and pathways as targets for therapeutic intervention
US20100144838A1 (en) * 2008-12-01 2010-06-10 Beck William T Methods for Identifying Modulators of Pyrimidine Tract Binding Protein
CN102858985A (zh) * 2009-07-24 2013-01-02 西格马-奥尔德里奇有限责任公司 基因组编辑方法
US20130209463A1 (en) * 2010-06-30 2013-08-15 Compugen Ltd. Polypeptides and uses thereof as a drug for treatment of multiple sclerosis, rheumatoid arthritis and other autoimmune disorders
WO2014071157A1 (en) * 2012-11-01 2014-05-08 The Regents Of The University Of California Methods for engineering non-neuronal cells into neurons and using newly engineered neurons to treat neurodegenerative diseases
WO2017003466A1 (en) * 2015-06-30 2017-01-05 University Of South Florida Conversion of non-neuronal cells into neurons
CN105950557B (zh) * 2016-04-22 2019-10-25 中国科学院生物物理研究所 一种控制人神经细胞重编程的信号通路及其应用
WO2018048877A1 (en) * 2016-09-06 2018-03-15 The University Of North Carolina At Chapel Hill Generation of neurons by reprogramming of oligodendrocytes and oligodendrocyte precursor cells
WO2018112446A2 (en) * 2016-12-18 2018-06-21 Selonterra, Inc. Use of apoe4 motif-mediated genes for diagnosis and treatment of alzheimer's disease
CA3096691A1 (en) * 2018-04-11 2019-10-17 The Regents Of The University Of California Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders
WO2020113338A1 (en) * 2018-12-05 2020-06-11 Adaerata, Limited Partnership Method of reducing neuronal microtubule binding protein tau (tau) levels
CN109913420A (zh) * 2019-03-07 2019-06-21 北京师范大学 Cdc20共表达基因网络作为胶质瘤治疗靶点的应用
CN109777877B (zh) * 2019-03-19 2022-01-04 宁波市第一医院 基于ptbp1甲基化辅助诊断脑动脉瘤的检测试剂盒及其应用

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EP4039801A1 (en) 2022-08-10
WO2021032069A1 (en) 2021-02-25
US20220273726A1 (en) 2022-09-01
WO2021032068A1 (zh) 2021-02-25
US20250345365A1 (en) 2025-11-13
EP4039801A4 (en) 2023-10-18

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