CN113646006A - 用于营养不良型大疱性表皮松解症的治疗剂 - Google Patents

用于营养不良型大疱性表皮松解症的治疗剂 Download PDF

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CN113646006A
CN113646006A CN202080022396.3A CN202080022396A CN113646006A CN 113646006 A CN113646006 A CN 113646006A CN 202080022396 A CN202080022396 A CN 202080022396A CN 113646006 A CN113646006 A CN 113646006A
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cells
cell
composition
acid sequence
mesenchymal stem
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玉井克人
菊池康
玉越智树
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University of Osaka NUC
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
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    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/39—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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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
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0662—Stem cells
    • C12N5/0663—Bone marrow mesenchymal stem cells (BM-MSC)
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00—Genetically modified cells

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CN202080022396.3A 2019-01-18 2020-01-17 用于营养不良型大疱性表皮松解症的治疗剂 Pending CN113646006A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2019-007201 2019-01-18
JP2019007201 2019-01-18
PCT/JP2020/001433 WO2020149395A1 (ja) 2019-01-18 2020-01-17 栄養障害型表皮水疱症治療薬

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US (1) US20220088083A1 (https=)
EP (1) EP3912644A4 (https=)
JP (2) JP7536296B2 (https=)
KR (1) KR20210116531A (https=)
CN (1) CN113646006A (https=)
AU (1) AU2020209687A1 (https=)
WO (1) WO2020149395A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
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CN116555349A (zh) * 2023-01-09 2023-08-08 中吉智药(南京)生物技术有限公司 一种腺相关病毒载体及其构建方法与应用
CN118001404A (zh) * 2024-02-06 2024-05-10 四川大学华西医院 抑制C17orf67基因表达试剂在制备治疗获得性大疱表皮松解症药物中的用途

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AU2020459825A1 (en) * 2020-07-22 2023-03-16 Osaka University Therapeutic agent for dystrophic epidermolysis bullosa
JP2025094280A (ja) * 2022-01-21 2025-06-25 国立大学法人大阪大学 細胞シート
KR102736437B1 (ko) * 2024-06-20 2024-12-02 주식회사 에이바이오테크 콜라겐 합성 촉진 활성을 갖는 콜라겐 타입 7 저분자 펩타이드 및 이를 이용한 방법

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CN105683376A (zh) * 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
WO2017161180A1 (en) * 2016-03-18 2017-09-21 Intrexon Corporation Compositions and methods for treatment of type vii collagen deficiencies
WO2018007871A1 (en) * 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
CN107904208A (zh) * 2017-12-25 2018-04-13 云舟生物科技（广州）有限公司 细胞表型研究用的细胞克隆及其筛选方法和应用
CN109072255A (zh) * 2016-04-08 2018-12-21 克里斯托生物技术股份有限公司 用于治疗皮肤的伤口、病症和疾病的组合物和方法

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WO2017161180A1 (en) * 2016-03-18 2017-09-21 Intrexon Corporation Compositions and methods for treatment of type vii collagen deficiencies
CN109072255A (zh) * 2016-04-08 2018-12-21 克里斯托生物技术股份有限公司 用于治疗皮肤的伤口、病症和疾病的组合物和方法
WO2018007871A1 (en) * 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
CN107904208A (zh) * 2017-12-25 2018-04-13 云舟生物科技（广州）有限公司 细胞表型研究用的细胞克隆及其筛选方法和应用

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116555349A (zh) * 2023-01-09 2023-08-08 中吉智药(南京)生物技术有限公司 一种腺相关病毒载体及其构建方法与应用
CN118001404A (zh) * 2024-02-06 2024-05-10 四川大学华西医院 抑制C17orf67基因表达试剂在制备治疗获得性大疱表皮松解症药物中的用途

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US20220088083A1 (en) 2022-03-24
EP3912644A4 (en) 2022-09-28
AU2020209687A1 (en) 2021-07-29
JP2024153826A (ja) 2024-10-29
JP7833204B2 (ja) 2026-03-19
WO2020149395A1 (ja) 2020-07-23
JPWO2020149395A1 (ja) 2021-11-25
KR20210116531A (ko) 2021-09-27
JP7536296B2 (ja) 2024-08-20
EP3912644A1 (en) 2021-11-24

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