CA3096691A1 - 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
CA3096691A1
CA3096691A1 CA3096691A CA3096691A CA3096691A1 CA 3096691 A1 CA3096691 A1 CA 3096691A1 CA 3096691 A CA3096691 A CA 3096691A CA 3096691 A CA3096691 A CA 3096691A CA 3096691 A1 CA3096691 A1 CA 3096691A1
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cell
ptb
neuronal
neuron
human
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French (fr)
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Xiang-Dong Fu
Hao Qian
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University of California San Diego UCSD
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University of California San Diego UCSD
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    • 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
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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
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    • 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
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    • 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
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    • C12N5/0619—Neurons
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    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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    • 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)
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  • Plant Pathology (AREA)
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  • Hospice & Palliative Care (AREA)
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  • 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)
CA3096691A 2018-04-11 2019-04-11 Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders Pending CA3096691A1 (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
CA3096691A1 true CA3096691A1 (en) 2019-10-17

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CA3096691A Pending CA3096691A1 (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 (enExample)
EP (1) EP3773749A4 (enExample)
JP (1) JP2021520200A (enExample)
KR (1) KR20200142537A (enExample)
CN (1) CN112312928A (enExample)
AU (1) AU2019252534A1 (enExample)
CA (1) CA3096691A1 (enExample)
GB (2) GB2588307B (enExample)
WO (1) WO2019200129A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021031565A1 (en) * 2019-08-16 2021-02-25 Center For Excellence In Brain Science And Intelligence Technology, Chinese Academy Of Sciences Treatment of Neuronal Diseases
EP4039801A4 (en) * 2019-08-16 2023-10-18 Center For Excellence In Brain Science And Intelligence Technology, Chinese Academy Of Sciences USE OF PTBP1 INHIBITORS TO PREVENT AND/OR TREAT NERVOUS SYSTEM DISEASES ASSOCIATED WITH FUNCTIONAL NEURONAL DEATH
WO2021232062A1 (en) * 2020-05-13 2021-11-18 Figene, Llc Fibroblast based therapy for treatment of parkinson's disease
EP4267196A4 (en) * 2020-12-25 2024-03-20 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
CN116218774A (zh) * 2021-03-26 2023-06-06 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
US20240207324A1 (en) * 2021-04-21 2024-06-27 Shanghai Unixell Biotechnology Co., Ltd. Method for quality control and enrichment of human dopaminergic neural precursor cells
CN115887655B (zh) * 2021-09-30 2025-06-17 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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011091048A1 (en) 2010-01-19 2011-07-28 The Board Of Trustees Of The Leland Stanford Junior University Direct conversion of cells to cells of other lineages
EP2542267A4 (en) * 2010-03-05 2014-09-17 Univ Columbia THE RNA BINDEPROTEIN HNRNPA1, HNRNPA2 AND PTB-INHIBITING RNAS AND USES THEREOF
WO2012118988A1 (en) 2011-03-01 2012-09-07 The Scripps Research Institute Direct reprogramming of human fibroblasts to functional neurons under defined conditions
WO2014015261A1 (en) * 2012-07-19 2014-01-23 The Penn State Research Foundation Regenerating functional neurons for treatment of disease and injury in 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
WO2016086092A1 (en) * 2014-11-25 2016-06-02 The Penn State Research Foundation Chemical reprogramming of human glial cells into neurons for brain and spinal cord repair
US9481864B1 (en) * 2015-06-30 2016-11-01 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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JP2021520200A (ja) 2021-08-19
US11702656B2 (en) 2023-07-18
WO2019200129A1 (en) 2019-10-17
GB2588307A (en) 2021-04-21
EP3773749A1 (en) 2021-02-17
CN112312928A (zh) 2021-02-02
GB2588307B (en) 2023-08-16
GB202017804D0 (en) 2020-12-23
KR20200142537A (ko) 2020-12-22
AU2019252534A1 (en) 2020-10-29
US20200377891A1 (en) 2020-12-03
US20240218362A1 (en) 2024-07-04
GB202017810D0 (en) 2020-12-23
EP3773749A4 (en) 2021-12-22

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