CN112312928A - 非神经元细胞向神经元的重编程、以及治疗神经退行性疾病和病症的方法和组合物 - Google Patents

非神经元细胞向神经元的重编程、以及治疗神经退行性疾病和病症的方法和组合物 Download PDF

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CN112312928A
CN112312928A CN201980039567.0A CN201980039567A CN112312928A CN 112312928 A CN112312928 A CN 112312928A CN 201980039567 A CN201980039567 A CN 201980039567A CN 112312928 A CN112312928 A CN 112312928A
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cells
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X·D·付
H·钱
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University of California San Diego UCSD
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CN201980039567.0A 2018-04-11 2019-04-11 非神经元细胞向神经元的重编程、以及治疗神经退行性疾病和病症的方法和组合物 Pending CN112312928A (zh)

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Application Number Priority Date Filing Date Title
US201862656322P 2018-04-11 2018-04-11
US62/656322 2018-04-11
US201862718774P 2018-08-14 2018-08-14
US62/718774 2018-08-14
PCT/US2019/027027 WO2019200129A1 (en) 2018-04-11 2019-04-11 Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders

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CN (1) CN112312928A (https=)
AU (1) AU2019252534A1 (https=)
CA (1) CA3096691A1 (https=)
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Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112386699A (zh) * 2019-08-16 2021-02-23 中国科学院脑科学与智能技术卓越创新中心 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
CN112941019A (zh) * 2021-03-18 2021-06-11 浙江大学 一种促进体细胞重编程的方法
CN113171369A (zh) * 2021-04-16 2021-07-27 南通大学 多聚嘧啶序列结合蛋白在制备脊髓损伤的修复药物中的应用
WO2022171167A1 (zh) * 2021-02-10 2022-08-18 中国科学院脑科学与智能技术卓越创新中心 胶质细胞向神经元转分化用于预防或治疗神经元功能缺失或死亡相关疾病
WO2022222974A1 (zh) * 2021-04-21 2022-10-27 上海跃赛生物科技有限公司 一种质控和富集人多巴胺能神经前体细胞的方法
CN115887655A (zh) * 2021-09-30 2023-04-04 中国科学院脑科学与智能技术卓越创新中心 直接转分化治疗神经系统疾病
CN116144596A (zh) * 2021-03-26 2023-05-23 广西大学 一种体外高效诱导人体细胞重编程为神经元细胞的方法

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022543722A (ja) * 2019-08-16 2022-10-14 センター フォー エクセレンス イン ブレイン サイエンス アンド インテリジェンス テクノロジー，チャイニーズ アカデミー オブ サイエンシーズ 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
US20230242873A1 (en) * 2020-05-13 2023-08-03 Figene, Llc Fibroblast based therapy for treatment of parkinson's disease
US20240050589A1 (en) * 2020-12-25 2024-02-15 Shanghai Genemagic Biosciences Co., Ltd. Treatment of neurological diseases
CN112661828B (zh) * 2021-01-15 2022-04-01 武汉大学 一种突触素的抗原肽及其抗体与应用
KR102712694B1 (ko) * 2021-10-20 2024-10-02 서울시립대학교 산학협력단 자가조립된 rna 구조체를 포함하는 직접교차분화 유도용 조성물

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150250900A1 (en) * 2012-07-19 2015-09-10 The Penn State Research Foundation Regenerating functional neurons for treatment of disease and injury in the nervous system
US20150299698A1 (en) * 2012-11-01 2015-10-22 The Regents Of The University Of California Methods for engineering non-neuronal cells into neurons and using newly engineered neurons to treat neurodegenerative diseases
CN105950557A (zh) * 2016-04-22 2016-09-21 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
US20170002317A1 (en) * 2015-06-30 2017-01-05 University Of South Florida Conversion of non-neuronal cells into neurons

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9057053B2 (en) 2010-01-19 2015-06-16 The Board Of Trustees Of The Leland Stanford Junior University Direct conversion of cells to cells of other lineages
US9206426B2 (en) * 2010-03-05 2015-12-08 The Trustees Of Columbia University In The City Of New York Inhibitory RNAs to RNA binding proteins hnRNPA1, hnRNPA2 and PTB and uses thereof
US20140051085A1 (en) 2011-03-01 2014-02-20 The Scripps Research Institute Direct reprogramming of human fibroblasts to functional neurons under defined conditions
US9730975B2 (en) * 2014-11-25 2017-08-15 The Penn State Research Foundation Chemical reprogramming of human glial cells into neurons for brain and spinal cord repair
US20180318260A1 (en) * 2015-11-04 2018-11-08 The Scripps Research Institute Compositions and methods for treating cystic fibrosis

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150250900A1 (en) * 2012-07-19 2015-09-10 The Penn State Research Foundation Regenerating functional neurons for treatment of disease and injury in the nervous system
US20150299698A1 (en) * 2012-11-01 2015-10-22 The Regents Of The University Of California Methods for engineering non-neuronal cells into neurons and using newly engineered neurons to treat neurodegenerative diseases
US20170002317A1 (en) * 2015-06-30 2017-01-05 University Of South Florida Conversion of non-neuronal cells into neurons
CN105950557A (zh) * 2016-04-22 2016-09-21 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

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
DAMIER ET AL.: ""The substantia nigra of the human brain: II. Patterns of loss of dopamine-containing neurons in Parkinson’s diaease"", 《BRAIN》 *
DAOFANG DING, ET AL.: ""Mash1 efficiently reprograms rat astrocytes into neurons"", 《NEURAL REGENERATION RESEARCH》 *
XIN LIANG, ET AL.: ""Inhibition of polypyrimidine tract-binding protein 3 induces apoptosis and cell cycle arrest, and enhances the cytotoxicity of 5-fluotouracil in gastric cancer cells"", 《BRITISH JOURNAL OR CANCER》 *
YUANCHAO XUE, ET AL.: ""Sequential regulatory loops as key gatekeepers for neuronal reprogramming in human cells"", 《NATURE NEUROSCIENCE》 *

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112386699A (zh) * 2019-08-16 2021-02-23 中国科学院脑科学与智能技术卓越创新中心 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
WO2022171167A1 (zh) * 2021-02-10 2022-08-18 中国科学院脑科学与智能技术卓越创新中心 胶质细胞向神经元转分化用于预防或治疗神经元功能缺失或死亡相关疾病
CN112941019A (zh) * 2021-03-18 2021-06-11 浙江大学 一种促进体细胞重编程的方法
CN116144596A (zh) * 2021-03-26 2023-05-23 广西大学 一种体外高效诱导人体细胞重编程为神经元细胞的方法
CN113171369A (zh) * 2021-04-16 2021-07-27 南通大学 多聚嘧啶序列结合蛋白在制备脊髓损伤的修复药物中的应用
WO2022148500A3 (zh) * 2021-04-16 2022-08-25 南通大学 多聚嘧啶序列结合蛋白在制备脊髓损伤的修复药物中的应用
WO2022222974A1 (zh) * 2021-04-21 2022-10-27 上海跃赛生物科技有限公司 一种质控和富集人多巴胺能神经前体细胞的方法
CN115887655A (zh) * 2021-09-30 2023-04-04 中国科学院脑科学与智能技术卓越创新中心 直接转分化治疗神经系统疾病

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EP3773749A1 (en) 2021-02-17
US20240218362A1 (en) 2024-07-04
JP2021520200A (ja) 2021-08-19
US11702656B2 (en) 2023-07-18
US20200377891A1 (en) 2020-12-03
WO2019200129A1 (en) 2019-10-17
AU2019252534A1 (en) 2020-10-29
GB202017804D0 (en) 2020-12-23
KR20200142537A (ko) 2020-12-22
CA3096691A1 (en) 2019-10-17
GB2588307B (en) 2023-08-16
EP3773749A4 (en) 2021-12-22
GB2588307A (en) 2021-04-21

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