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 (https=
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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)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurosurgery (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 (https=) 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 (https=)
EP (1) EP4192960A4 (https=)
JP (1) JP7853952B2 (https=)
KR (1) KR20230043123A (https=)
CN (1) CN116113700A (https=)
AU (1) AU2021321412A1 (https=)
BR (1) BR112023001418A2 (https=)
CA (1) CA3184233A1 (https=)
IL (1) IL300185A (https=)
MX (1) MX2023001419A (https=)
WO (1) WO2022031760A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE055862T2 (hu) 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
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
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
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
WO2025082176A1 (zh) * 2023-10-19 2025-04-24 北京脑科学与类脑研究所 一种脑血管内皮细胞嵌合型启动子和重组表达载体及应用
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
MX2023001419A (es) 2023-05-16
IL300185A (en) 2023-03-01
KR20230043123A (ko) 2023-03-30
BR112023001418A2 (pt) 2023-03-07
CA3184233A1 (en) 2022-02-10
JP2023536902A (ja) 2023-08-30
EP4192960A4 (en) 2024-11-27
AU2021321412A1 (en) 2023-04-06
WO2022031760A1 (en) 2022-02-10
CN116113700A (zh) 2023-05-12
EP4192960A1 (en) 2023-06-14
US20230272422A1 (en) 2023-08-31

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