CN116113700A - 用于glut1表达的腺相关病毒载体及其用途 - Google Patents

用于glut1表达的腺相关病毒载体及其用途 Download PDF

Info

Publication number
CN116113700A
CN116113700A CN202180057450.2A CN202180057450A CN116113700A CN 116113700 A CN116113700 A CN 116113700A CN 202180057450 A CN202180057450 A CN 202180057450A CN 116113700 A CN116113700 A CN 116113700A
Authority
CN
China
Prior art keywords
gly
promoter
pro
leu
ser
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN202180057450.2A
Other languages
English (en)
Chinese (zh)
Inventor
C·D·赫尔佐格
C·B·萨克拉门多
R·普拉巴卡尔
D·里克斯
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Spaceship Seven LLC
Original Assignee
Spaceship Seven LLC
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Spaceship Seven LLC filed Critical Spaceship Seven LLC
Publication of CN116113700A publication Critical patent/CN116113700A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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

Landscapes

  • 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)
  • Biophysics (AREA)
  • 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)
CN202180057450.2A 2020-08-05 2021-08-03 用于glut1表达的腺相关病毒载体及其用途 Pending CN116113700A (zh)

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 (1)

Publication Number Publication Date
CN116113700A true CN116113700A (zh) 2023-05-12

Family

ID=80118621

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180057450.2A Pending CN116113700A (zh) 2020-08-05 2021-08-03 用于glut1表达的腺相关病毒载体及其用途

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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (7)

* 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
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

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8071740B2 (en) 2000-11-17 2011-12-06 Vascular Biogenics Ltd. Promoters exhibiting endothelial cell specificity and methods of using same for regulation of angiogenesis
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
KR20240119152A (ko) 2015-03-10 2024-08-06 더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕 재조합 glut1 아데노-관련 바이러스 벡터 작제물 및 glut1 발현을 회복하기 위한 관련된 방법
US11446375B2 (en) 2015-12-22 2022-09-20 Vrije Universiteit Brussel Endothelium-specific nucleic acid regulatory elements and methods and use thereof
JP6996728B2 (ja) * 2016-01-07 2022-01-17 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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

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
JP7853952B2 (ja) 2026-04-30
EP4192960A4 (en) 2024-11-27
AU2021321412A1 (en) 2023-04-06
WO2022031760A1 (en) 2022-02-10
EP4192960A1 (en) 2023-06-14
US20230272422A1 (en) 2023-08-31

Similar Documents

Publication Publication Date Title
CN116113700A (zh) 用于glut1表达的腺相关病毒载体及其用途
CN116194154A (zh) 使用aav载体的plakophilin-2(pkp2)基因疗法
CN112368390B (zh) Cns变性的基因疗法
AU2018375192B2 (en) Engineered DNA binding proteins
CN114174520B (zh) 用于选择性基因调节的组合物和方法
CN107849547B (zh) 深内含子突变的基因编辑
EP1696036B1 (en) Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
CN116171325A (zh) 用于eEF1A2的基因疗法载体及其用途
KR20230042468A (ko) Csrp3 (시스테인 및 글리신 풍부 단백질 3) 유전자 요법
KR20210131370A (ko) Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스
CN115151648A (zh) 用于治疗cdkl5缺陷障碍的基因疗法
CN116685329B (zh) 核酸构建体及其用于治疗脊髓性肌肉萎缩症的用途
KR20210144696A (ko) 라민병증 치료용 조성물 및 치료 방법
KR20230058102A (ko) Grn 관련 성인 발병 신경변성 치료를 위한 재조합 아데노-연관 바이러스
CN117545842A (zh) SMN1和miR-23a在治疗脊髓性肌萎缩中的协同效应
CN114402075A (zh) 乌谢尔综合征(ush2a)的基因疗法
KR20230019402A (ko) 프로그래뉼린 연관 신경변성 질환 또는 장애의 치료를 위한 아데노-연관 바이러스 (aav) 시스템
CN113874512A (zh) 诱导毛细胞分化的组合物和方法
CN112639108A (zh) 治疗非综合征性感觉神经性听力损失的方法
RU2742837C1 (ru) Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
KR20230003478A (ko) 비-바이러스성 dna 벡터 및 고셰 치료제 발현을 위한 이의 용도
CN112639107B (zh) 治疗clrn1相关的听力损失和/或视力损失的方法
KR20220007601A (ko) 치료제 투여를 위한 조성물 및 방법
RU2855847C1 (ru) Кассета экспрессии, вектор генной терапии синдрома дефицита glut1, способ лечения синдрома дефицита glut1, способ экспрессии glut1, фармацевтическая композиция и набор для экспрессии glut1
KR20220145838A (ko) Gm1 강글리오사이드증을 치료하는 데 유용한 조성물

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination