JP2022552003A - 脊髄損傷およびａｌｓの治療のための機能的ニューロンの再生 - Google Patents

脊髄損傷およびａｌｓの治療のための機能的ニューロンの再生 Download PDF

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JP2022552003A
JP2022552003A JP2022522966A JP2022522966A JP2022552003A JP 2022552003 A JP2022552003 A JP 2022552003A JP 2022522966 A JP2022522966 A JP 2022522966A JP 2022522966 A JP2022522966 A JP 2022522966A JP 2022552003 A JP2022552003 A JP 2022552003A
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nucleic acid
neurod1
polypeptide
biologically active
spinal cord
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JP2022552003A5 (https=
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チェン，ゴン
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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
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • A—HUMAN NECESSITIES
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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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
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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
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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
    • C—CHEMISTRY; METALLURGY
    • 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
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    • 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)
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  • 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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  • 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)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
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  • 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)
JP2022522966A 2019-10-17 2020-10-16 脊髄損傷およびａｌｓの治療のための機能的ニューロンの再生 Pending JP2022552003A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025124068A JP2025184866A (ja) 2019-10-17 2025-07-24 脊髄損傷およびａｌｓの治療のための機能的ニューロンの再生

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962916713P 2019-10-17 2019-10-17
US62/916,713 2019-10-17
US202063040989P 2020-06-18 2020-06-18
US63/040,989 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

Related Child Applications (1)

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JP2025124068A Division JP2025184866A (ja) 2019-10-17 2025-07-24 脊髄損傷およびａｌｓの治療のための機能的ニューロンの再生

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JP2022552003A true JP2022552003A (ja) 2022-12-14
JP2022552003A5 JP2022552003A5 (https=) 2023-10-20

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JP2022522966A Pending JP2022552003A (ja) 2019-10-17 2020-10-16 脊髄損傷およびａｌｓの治療のための機能的ニューロンの再生
JP2025124068A Pending JP2025184866A (ja) 2019-10-17 2025-07-24 脊髄損傷およびａｌｓの治療のための機能的ニューロンの再生

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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=)

Cited By (1)

* Cited by examiner, † Cited by third party
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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140024599A1 (en) * 2012-07-19 2014-01-23 The Penn State Research Foundation Methods and compositions for treatment of disease or injury of the nervous system
WO2014148646A1 (ja) * 2013-03-21 2014-09-25 国立大学法人京都大学 神経分化誘導用の多能性幹細胞
US20150023927A1 (en) * 2011-08-17 2015-01-22 Children's Medical Center Corporation Conversion of somatic cells into functional spinal motor neurons, and methods and uses thereof
WO2018051293A1 (en) * 2016-09-19 2018-03-22 Fondazione Istituto Italiano Di Tecnologia miRNA PHARMACEUTICAL COMPOSITION AND THERAPEUTIC USES THEREOF

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2054079A4 (en) * 2006-07-19 2009-12-23 Univ Florida COMPOSITIONS FOR CELL REPROGRAMMING AND USES THEREOF
WO2014201133A1 (en) * 2013-06-11 2014-12-18 President And Fellows Of Harvard College Compositions and methods for improving induced neuron generation
CA2934065C (en) * 2013-10-25 2023-05-09 Qurgen, Inc. Methods, systems and compositions relating to cell conversion via protein-induced in-vivo cell reprogramming
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
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
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
US20200377885A1 (en) * 2017-08-07 2020-12-03 Washington University Compositions and methods for the generation of neurons and uses thereof
WO2022072325A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Neurod1 combination vector

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150023927A1 (en) * 2011-08-17 2015-01-22 Children's Medical Center Corporation Conversion of somatic cells into functional spinal motor neurons, and methods and uses thereof
US20140024599A1 (en) * 2012-07-19 2014-01-23 The Penn State Research Foundation Methods and compositions for treatment of disease or injury of the nervous system
WO2014148646A1 (ja) * 2013-03-21 2014-09-25 国立大学法人京都大学 神経分化誘導用の多能性幹細胞
WO2018051293A1 (en) * 2016-09-19 2018-03-22 Fondazione Istituto Italiano Di Tecnologia miRNA PHARMACEUTICAL COMPOSITION AND THERAPEUTIC USES THEREOF

Cited By (1)

* Cited by examiner, † Cited by third party
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

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US20260021160A1 (en) 2026-01-22
WO2021076983A1 (en) 2021-04-22
EP4045148A4 (en) 2023-11-08
CN114364436A (zh) 2022-04-15
US20210162002A1 (en) 2021-06-03
AU2020365130A1 (en) 2022-05-12
EP4045148A1 (en) 2022-08-24
CA3157523A1 (en) 2021-04-22
JP2025184866A (ja) 2025-12-18
CN120899949A (zh) 2025-11-07

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