MX2017011615A - Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1. - Google Patents

Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1.

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Publication number
MX2017011615A
MX2017011615A MX2017011615A MX2017011615A MX2017011615A MX 2017011615 A MX2017011615 A MX 2017011615A MX 2017011615 A MX2017011615 A MX 2017011615A MX 2017011615 A MX2017011615 A MX 2017011615A MX 2017011615 A MX2017011615 A MX 2017011615A
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MX
Mexico
Prior art keywords
glut1
certain embodiments
relates
present
recombinant
Prior art date
Application number
MX2017011615A
Other languages
English (en)
Inventor
Gao Guangping
ENGELSTAD Kristin
De Vivo Darryl
Monani Umrao
Original Assignee
Univ Columbia
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Publication date
Application filed by Univ Columbia filed Critical Univ Columbia
Publication of MX2017011615A publication Critical patent/MX2017011615A/es

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N15/11—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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente invención se refiere a construcciones de vectores virales adeno-asociados (rAAV) de Glut1 recombinantes y métodos relacionados para restablecer la expresión de Glut1 en mamíferos con deficiencia de Glut1. En determinadas modalidades, el rAAV además comprende un promotor de ß-actina de pollo, en donde el rAAV es capaz de cruzar la barrera hematoencefálica (BBB). En determinadas modalidades, la presente invención se refiere a una composición que comprende cualquiera de los AAV recombinantes descritos en la presente. En determinadas modalidades, la presente invención se refiere a un kit que comprende una cubierta de un recipiente que comprende la composición descrita en la presente. En determinadas modalidades, la presente invención se refiere a métodos para restablecer el transporte de Glut1 en la BBB de un sujeto, que comprende administrarle al sujeto una cantidad eficaz de cualquiera de los vectores AAV recombinantes descritos en la presente. En determinadas modalidades, la presente invención se refiere a un método para tratar el síndrome de deficiencia de Glut1 en un sujeto que lo necesita.
MX2017011615A 2015-03-10 2016-03-10 Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1. MX2017011615A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562130899P 2015-03-10 2015-03-10
PCT/US2016/021810 WO2016145217A1 (en) 2015-03-10 2016-03-10 Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression

Publications (1)

Publication Number Publication Date
MX2017011615A true MX2017011615A (es) 2018-04-11

Family

ID=56879055

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017011615A MX2017011615A (es) 2015-03-10 2016-03-10 Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1.
MX2023001213A MX2023001213A (es) 2015-03-10 2017-09-08 Construcciones de vectores virales adeno-asociados de glut1 recombinantes y metodos relacionados para restablecer la expresion de glut1.

Family Applications After (1)

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MX2023001213A MX2023001213A (es) 2015-03-10 2017-09-08 Construcciones de vectores virales adeno-asociados de glut1 recombinantes y metodos relacionados para restablecer la expresion de glut1.

Country Status (20)

Country Link
US (2) US20180042991A1 (es)
EP (1) EP3268024B1 (es)
JP (3) JP2018509164A (es)
KR (1) KR20180016722A (es)
CN (1) CN107635575A (es)
AU (1) AU2016229000B2 (es)
BR (1) BR112017019294A2 (es)
CA (1) CA2978917A1 (es)
CL (1) CL2017002282A1 (es)
CO (1) CO2017010149A2 (es)
DK (1) DK3268024T3 (es)
EA (1) EA036051B1 (es)
ES (1) ES2836258T3 (es)
HU (1) HUE052577T2 (es)
IL (2) IL282053B2 (es)
MX (2) MX2017011615A (es)
PT (1) PT3268024T (es)
SG (1) SG11201707116QA (es)
UA (1) UA128249C2 (es)
WO (1) WO2016145217A1 (es)

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MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
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
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3098871A1 (en) * 2018-05-08 2019-11-14 Rutgers, The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
AU2019346655A1 (en) * 2018-09-28 2021-05-06 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
AU2020274339C1 (en) 2019-05-13 2023-08-31 Dna Twopointo Inc. Modifications of mammalian cells using artificial micro-RNA to alter their properties and the compositions of their products
CN114555814A (zh) * 2019-09-13 2022-05-27 罗特格斯新泽西州立大学 Aav相容的层粘连蛋白-连接子聚合蛋白
EP4142775A1 (en) * 2020-05-01 2023-03-08 Atlas Biotechnology S.A. Treatment and/or prevention of a disease or a syndrome related to a virus infection
US20230248824A1 (en) * 2020-07-09 2023-08-10 The Board of Trutees of the Leland Stanford Junior University Immune cells with increased glycolytic flux
CA3184233A1 (en) * 2020-08-05 2022-02-10 Christopher Dean HERZOG Adeno-associated viral vector for glut1 expression and uses thereof
EP4192487A1 (en) 2020-08-07 2023-06-14 Spacecraft Seven, LLC Plakophilin-2 (pkp2) gene therapy using aav vector
US20230313187A1 (en) * 2020-09-04 2023-10-05 Dna Twopointo Inc. Modifications of mammalian cells using artificial micro-rna to alter their properties and the compositions of their products
WO2023154763A2 (en) * 2022-02-08 2023-08-17 Spacecraft Seven, Llc Adeno-associated viral vector for glut1 expression and uses thereof

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Also Published As

Publication number Publication date
EA036051B1 (ru) 2020-09-18
JP2018509164A (ja) 2018-04-05
CL2017002282A1 (es) 2018-04-13
WO2016145217A1 (en) 2016-09-15
AU2016229000B2 (en) 2021-11-04
KR20180016722A (ko) 2018-02-19
EP3268024A1 (en) 2018-01-17
JP2023144087A (ja) 2023-10-06
BR112017019294A2 (pt) 2018-05-02
UA128249C2 (uk) 2024-05-22
CO2017010149A2 (es) 2018-02-28
IL282053B2 (en) 2023-03-01
MX2023001213A (es) 2023-03-03
HUE052577T2 (hu) 2021-05-28
IL282053B (en) 2022-11-01
IL254286A0 (en) 2017-10-31
ES2836258T3 (es) 2021-06-24
IL254286B (en) 2021-04-29
US20180042991A1 (en) 2018-02-15
AU2016229000A1 (en) 2017-09-21
IL282053A (en) 2021-05-31
EA201791936A8 (ru) 2018-11-30
DK3268024T3 (da) 2020-12-07
SG11201707116QA (en) 2017-09-28
EP3268024B1 (en) 2020-09-09
US20210069292A1 (en) 2021-03-11
JP2021097689A (ja) 2021-07-01
EP3268024A4 (en) 2018-08-01
PT3268024T (pt) 2020-12-09
EA201791936A1 (ru) 2018-01-31
CA2978917A1 (en) 2016-09-15
CN107635575A (zh) 2018-01-26

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