AU2019252534A1 - Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders - Google Patents

Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders Download PDF

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Publication number
AU2019252534A1
AU2019252534A1 AU2019252534A AU2019252534A AU2019252534A1 AU 2019252534 A1 AU2019252534 A1 AU 2019252534A1 AU 2019252534 A AU2019252534 A AU 2019252534A AU 2019252534 A AU2019252534 A AU 2019252534A AU 2019252534 A1 AU2019252534 A1 AU 2019252534A1
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cell
ptb
neuronal
neuron
human
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AU2019252534A
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Inventor
Xiang-Dong Fu
Hao Qian
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University of California San Diego UCSD
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University of California Berkeley
University of California San Diego UCSD
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0622—Glial cells, e.g. astrocytes, oligodendrocytes; Schwann cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0619—Neurons
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/999—Small molecules not provided for elsewhere
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Cell Biology (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2019252534A 2018-04-11 2019-04-11 Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders Abandoned AU2019252534A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862656322P 2018-04-11 2018-04-11
US62/656,322 2018-04-11
US201862718774P 2018-08-14 2018-08-14
US62/718,774 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

Publications (1)

Publication Number Publication Date
AU2019252534A1 true AU2019252534A1 (en) 2020-10-29

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AU2019252534A Abandoned AU2019252534A1 (en) 2018-04-11 2019-04-11 Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders

Country Status (9)

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US (2) US11702656B2 (https=)
EP (1) EP3773749A4 (https=)
JP (1) JP2021520200A (https=)
KR (1) KR20200142537A (https=)
CN (1) CN112312928A (https=)
AU (1) AU2019252534A1 (https=)
CA (1) CA3096691A1 (https=)
GB (2) GB202017810D0 (https=)
WO (1) WO2019200129A1 (https=)

Families Citing this family (12)

* 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
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
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
CN117279668A (zh) * 2021-02-10 2023-12-22 上海鲸奇生物科技有限公司 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
CN112941019B (zh) * 2021-03-18 2023-01-06 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
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
CN120754249A (zh) * 2021-09-30 2025-10-10 上海鲸奇生物科技有限公司 直接转分化治疗神经系统疾病
KR102712694B1 (ko) * 2021-10-20 2024-10-02 서울시립대학교 산학협력단 자가조립된 rna 구조체를 포함하는 직접교차분화 유도용 조성물

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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
US10561742B2 (en) * 2012-07-19 2020-02-18 The Penn State Research Foundation Methods and compositions for treatment of disease or injury of the nervous system
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
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
WO2017003466A1 (en) * 2015-06-30 2017-01-05 University Of South Florida Conversion of non-neuronal cells into neurons
US20180318260A1 (en) * 2015-11-04 2018-11-08 The Scripps Research Institute Compositions and methods for treating cystic fibrosis
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

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Publication number Publication date
GB202017810D0 (en) 2020-12-23
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
GB202017804D0 (en) 2020-12-23
CN112312928A (zh) 2021-02-02
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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