JP7853952B2 - Ｇｌｕｔ１発現のためのアデノ随伴ウイルスベクターおよびその使用 - Google Patents

Ｇｌｕｔ１発現のためのアデノ随伴ウイルスベクターおよびその使用

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JP7853952B2
JP7853952B2 JP2023507555A JP2023507555A JP7853952B2 JP 7853952 B2 JP7853952 B2 JP 7853952B2 JP 2023507555 A JP2023507555 A JP 2023507555A JP 2023507555 A JP2023507555 A JP 2023507555A JP 7853952 B2 JP7853952 B2 JP 7853952B2
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vector
promoter
glut1
aav
pharmaceutical composition
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JP2023536902A (ja
JP2023536902A5 (OSRAM
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クリストファー ディーン ハーツォグ
チェスター ビッテンコート サクラメント
ラジ プラバカール
デイビッド リックス
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スペースクラフト セブン リミテッド ライアビリティ カンパニー
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    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A01K2217/075—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
    • 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
    • A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
JP2023507555A 2020-08-05 2021-08-03 Ｇｌｕｔ１発現のためのアデノ随伴ウイルスベクターおよびその使用 Active JP7853952B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063061726P 2020-08-05 2020-08-05
US63/061,726 2020-08-05
PCT/US2021/044416 WO2022031760A1 (en) 2020-08-05 2021-08-03 Adeno-associated viral vector for glut1 expression and uses thereof

Publications (3)

Publication Number Publication Date
JP2023536902A JP2023536902A (ja) 2023-08-30
JP2023536902A5 JP2023536902A5 (OSRAM) 2024-08-13
JP7853952B2 true JP7853952B2 (ja) 2026-04-30

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JP2023507555A Active JP7853952B2 (ja) 2020-08-05 2021-08-03 Ｇｌｕｔ１発現のためのアデノ随伴ウイルスベクターおよびその使用

Country Status (11)

Country Link
US (1) US20230272422A1 (OSRAM)
EP (1) EP4192960A4 (OSRAM)
JP (1) JP7853952B2 (OSRAM)
KR (1) KR20230043123A (OSRAM)
CN (1) CN116113700A (OSRAM)
AU (1) AU2021321412A1 (OSRAM)
BR (1) BR112023001418A2 (OSRAM)
CA (1) CA3184233A1 (OSRAM)
IL (1) IL300185A (OSRAM)
MX (1) MX2023001419A (OSRAM)
WO (1) WO2022031760A1 (OSRAM)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109641063B (zh) 2016-04-20 2022-11-15 能源环境和技术研究中心O.A., M.P. 用于增强pklr的基因表达的组合物和方法
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
JP7576754B2 (ja) 2018-04-11 2024-11-01 スペースクラフト セブン リミテッド ライアビリティ カンパニー 幹細胞移植のための組成物および方法
AU2019261646A1 (en) 2018-04-27 2020-11-12 Spacecraft Seven, Llc Gene therapy for CNS degeneration
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector
CN114457045B (zh) * 2022-02-25 2023-07-14 中国人民解放军军事科学院军事医学研究院 抑制Slc2a1的RNAi腺相关病毒及其制备和应用
CN119857100B (zh) * 2023-10-19 2025-12-09 北京脑科学与类脑研究所 一种小分子药物plx4032在制备脑动静脉畸形疾病治疗的药物中的应用
WO2025137149A1 (en) * 2023-12-19 2025-06-26 The Trustees Of Columbia University In The City Of New York Compositions and methods for therapeutic vectors targeting brain microvasculature

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008519596A (ja) 2004-11-14 2008-06-12 ヴァスキュラー バイオジェニックス リミテッド 内皮細胞特異性を示すプロモーター及びそのプロモーターを使って血管形成を制御する方法
JP2017123840A (ja) 2016-01-07 2017-07-20 学校法人自治医科大学 グルコーストランスポーター１発現用アデノ随伴ウイルスベクター
JP2018509164A (ja) 2015-03-10 2018-04-05 ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク 組換えＧｌｕｔ１アデノ随伴ウイルスベクターコンストラクトおよびＧｌｕｔ１発現を回復させる方法
JP2018538003A (ja) 2015-12-22 2018-12-27 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ 内皮特異的核酸調節エレメントならびにその方法および使用

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070161031A1 (en) * 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008519596A (ja) 2004-11-14 2008-06-12 ヴァスキュラー バイオジェニックス リミテッド 内皮細胞特異性を示すプロモーター及びそのプロモーターを使って血管形成を制御する方法
JP2018509164A (ja) 2015-03-10 2018-04-05 ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク 組換えＧｌｕｔ１アデノ随伴ウイルスベクターコンストラクトおよびＧｌｕｔ１発現を回復させる方法
JP2018538003A (ja) 2015-12-22 2018-12-27 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ 内皮特異的核酸調節エレメントならびにその方法および使用
JP2017123840A (ja) 2016-01-07 2017-07-20 学校法人自治医科大学 グルコーストランスポーター１発現用アデノ随伴ウイルスベクター

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Hypertension，2001年，vol. 38，p.65-70

Also Published As

Publication number Publication date
IL300185A (en) 2023-03-01
US20230272422A1 (en) 2023-08-31
JP2023536902A (ja) 2023-08-30
EP4192960A1 (en) 2023-06-14
BR112023001418A2 (pt) 2023-03-07
EP4192960A4 (en) 2024-11-27
WO2022031760A1 (en) 2022-02-10
KR20230043123A (ko) 2023-03-30
CA3184233A1 (en) 2022-02-10
AU2021321412A1 (en) 2023-04-06
MX2023001419A (es) 2023-05-16
CN116113700A (zh) 2023-05-12

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