BR112023001418A2 - Vetor viral adeno-associado para expressão de glut1 e seus usos - Google Patents

Vetor viral adeno-associado para expressão de glut1 e seus usos

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Publication number
BR112023001418A2
BR112023001418A2 BR112023001418A BR112023001418A BR112023001418A2 BR 112023001418 A2 BR112023001418 A2 BR 112023001418A2 BR 112023001418 A BR112023001418 A BR 112023001418A BR 112023001418 A BR112023001418 A BR 112023001418A BR 112023001418 A2 BR112023001418 A2 BR 112023001418A2
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BR
Brazil
Prior art keywords
adeno
glut1
viral vector
associated viral
raav
Prior art date
Application number
BR112023001418A
Other languages
English (en)
Inventor
Dean Herzog Christopher
Bittencort Sacramento Chester
Prabhakar Raj
Ricks David
Original Assignee
Spacecraft 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 Spacecraft Seven Llc filed Critical Spacecraft Seven Llc
Publication of BR112023001418A2 publication Critical patent/BR112023001418A2/pt

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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
    • 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
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    • C07—ORGANIC CHEMISTRY
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    • 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
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    • 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)
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  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • Organic Chemistry (AREA)
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  • 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)
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  • Plant Pathology (AREA)
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  • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)

Abstract

VETOR VIRAL ADENO-ASSOCIADO PARA EXPRESSÃO DE GLUT1 E SEUS USOS. Neste documento, é fornecida uma terapia gênica para a Síndrome de Deficiência de GLUT1 e distúrbios relacionados usando um vírion do vírus adeno-associado recombinante (rAAV) como um vetor para expressar uma proteína GLUT1 ou uma variante funcional desta. O vírion de rAAV pode usar um promotor endotelial específico, por exemplo, um promotor de FLT-1 ou Tie-1. O capsídeo pode ser um capsídeo AAV6, AA8, AAV9, AAVrh.74 ou AAVrh.10 ou uma variante funcional deste. Outros promotores ou capsídeos podem ser usados. São fornecidos ainda métodos de tratamento, tais como por via intracerebral e/ou intravenosa do vírion de rAAV, e outras composições e métodos.
BR112023001418A 2020-08-05 2021-08-03 Vetor viral adeno-associado para expressão de glut1 e seus usos BR112023001418A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063061726P 2020-08-05 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
BR112023001418A2 true BR112023001418A2 (pt) 2023-03-07

Family

ID=80118621

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023001418A BR112023001418A2 (pt) 2020-08-05 2021-08-03 Vetor viral adeno-associado para expressão de glut1 e seus usos

Country Status (11)

Country Link
US (1) US20230272422A1 (pt)
EP (1) EP4192960A1 (pt)
JP (1) JP2023536902A (pt)
KR (1) KR20230043123A (pt)
CN (1) CN116113700A (pt)
AU (1) AU2021321412A1 (pt)
BR (1) BR112023001418A2 (pt)
CA (1) CA3184233A1 (pt)
IL (1) IL300185A (pt)
MX (1) MX2023001419A (pt)
WO (1) WO2022031760A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2023001615A (es) 2020-08-07 2023-03-08 Spacecraft Seven Llc Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.
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

Family Cites Families (3)

* 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
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

Also Published As

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

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