CN114364436A - 再生功能神经元以用于治疗脊髓损伤和als - Google Patents

再生功能神经元以用于治疗脊髓损伤和als Download PDF

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Publication number
CN114364436A
CN114364436A CN202080060115.3A CN202080060115A CN114364436A CN 114364436 A CN114364436 A CN 114364436A CN 202080060115 A CN202080060115 A CN 202080060115A CN 114364436 A CN114364436 A CN 114364436A
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nucleic acid
polypeptide
neurod1
biologically active
active fragment
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Chinese (zh)
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陈功
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Penn State Research Foundation
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Penn State Research Foundation
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • C12N15/63—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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • 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
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Cell Biology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202080060115.3A 2019-10-17 2020-10-16 再生功能神经元以用于治疗脊髓损伤和als Pending CN114364436A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202511095971.3A CN120899949A (zh) 2019-10-17 2020-10-16 再生功能神经元以用于治疗脊髓损伤和als

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US201962916713P 2019-10-17 2019-10-17
US62/916713 2019-10-17
US202063040989P 2020-06-18 2020-06-18
US63/040989 2020-06-18
PCT/US2020/056108 WO2021076983A1 (en) 2019-10-17 2020-10-16 Regenerating functional neurons for treatment of spinal cord injury and als

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US (2) US20210162002A1 (https=)
EP (1) EP4045148A4 (https=)
JP (2) JP2022552003A (https=)
CN (2) CN114364436A (https=)
AU (1) AU2020365130A1 (https=)
CA (1) CA3157523A1 (https=)
WO (1) WO2021076983A1 (https=)

Families Citing this family (1)

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Publication number Priority date Publication date Assignee Title
WO2025143118A1 (ja) * 2023-12-27 2025-07-03 アステラス製薬株式会社 脊髄損傷の治療用医薬組成物及び脊髄損傷の非ヒト霊長類動物モデルの作製方法

Citations (5)

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CN101553245A (zh) * 2006-07-19 2009-10-07 佛罗里达大学研究基金会有限公司 用于细胞重编程的组合物及其用途
WO2013025963A2 (en) * 2011-08-17 2013-02-21 President And Fellows Of Harvard College Conversion of somatic cells into functional spinal motor neurons, and methods and uses thereof
CN104870634A (zh) * 2012-07-19 2015-08-26 宾夕法尼亚州研究基金会 再生用于在神经系统中治疗疾病和损伤的功能性神经元
CN107208045A (zh) * 2014-11-25 2017-09-26 宾州研究基金会 人神经胶质细胞转化成用于大脑和脊髓修复的神经细胞的化学重编程
US20190000982A1 (en) * 2013-10-25 2019-01-03 Qurgen, Inc. Methods, systems and compositions relating to cell conversion via protein-induced in-vivo cell reprogramming

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JP6473077B2 (ja) * 2013-03-21 2019-02-20 国立大学法人京都大学 神経分化誘導用の多能性幹細胞
WO2014201133A1 (en) * 2013-06-11 2014-12-18 President And Fellows Of Harvard College Compositions and methods for improving induced neuron generation
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
US10973930B2 (en) * 2016-02-18 2021-04-13 The Penn State Research Foundation Generating GABAergic neurons in brains
IT201600093825A1 (it) * 2016-09-19 2018-03-19 Fondazione St Italiano Tecnologia Composizione farmaceutica di miRNA e suoi usi terapeutici.
EP3589307B1 (en) * 2017-02-28 2022-02-16 The Penn State Research Foundation Regenerating functional neurons for treatment of neural injury caused by disruption of blood flow
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CN101553245A (zh) * 2006-07-19 2009-10-07 佛罗里达大学研究基金会有限公司 用于细胞重编程的组合物及其用途
WO2013025963A2 (en) * 2011-08-17 2013-02-21 President And Fellows Of Harvard College Conversion of somatic cells into functional spinal motor neurons, and methods and uses thereof
CN104870634A (zh) * 2012-07-19 2015-08-26 宾夕法尼亚州研究基金会 再生用于在神经系统中治疗疾病和损伤的功能性神经元
US20190000982A1 (en) * 2013-10-25 2019-01-03 Qurgen, Inc. Methods, systems and compositions relating to cell conversion via protein-induced in-vivo cell reprogramming
CN107208045A (zh) * 2014-11-25 2017-09-26 宾州研究基金会 人神经胶质细胞转化成用于大脑和脊髓修复的神经细胞的化学重编程

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Also Published As

Publication number Publication date
US20260021160A1 (en) 2026-01-22
WO2021076983A1 (en) 2021-04-22
EP4045148A4 (en) 2023-11-08
US20210162002A1 (en) 2021-06-03
AU2020365130A1 (en) 2022-05-12
EP4045148A1 (en) 2022-08-24
JP2022552003A (ja) 2022-12-14
CA3157523A1 (en) 2021-04-22
JP2025184866A (ja) 2025-12-18
CN120899949A (zh) 2025-11-07

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