GB2588307B - 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
GB2588307B
GB2588307B GB2017804.2A GB202017804A GB2588307B GB 2588307 B GB2588307 B GB 2588307B GB 202017804 A GB202017804 A GB 202017804A GB 2588307 B GB2588307 B GB 2588307B
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Prior art keywords
reprogramming
neurons
disorders
compositions
methods
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Expired - Fee Related
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GB2588307A (en
GB202017804D0 (en
Inventor
Fu Xiang-Dong
Qian Hao
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University of California Berkeley
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
    • 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
    • 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
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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
    • 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
    • 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
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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
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    • C—CHEMISTRY; METALLURGY
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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
    • C12N5/0619—Neurons
    • 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
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    • C12N2310/50—Physical structure
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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)
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  • 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)
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  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
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  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Hospice & Palliative Care (AREA)
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  • 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)
GB2017804.2A 2018-04-11 2019-04-11 Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders Expired - Fee Related GB2588307B (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862656322P 2018-04-11 2018-04-11
US201862718774P 2018-08-14 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 (3)

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GB202017804D0 GB202017804D0 (en) 2020-12-23
GB2588307A GB2588307A (en) 2021-04-21
GB2588307B true GB2588307B (en) 2023-08-16

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GBGB2017810.9A Ceased GB202017810D0 (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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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)

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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

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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
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WO2017079547A2 (en) * 2015-11-04 2017-05-11 The Scripps Research Institute Compositions and methods for treating cystic fibrosis

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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
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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
CA3096691A1 (en) 2019-10-17
CN112312928A (zh) 2021-02-02
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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