CO2017010149A2 - 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

Info

Publication number
CO2017010149A2
CO2017010149A2 CONC2017/0010149A CO2017010149A CO2017010149A2 CO 2017010149 A2 CO2017010149 A2 CO 2017010149A2 CO 2017010149 A CO2017010149 A CO 2017010149A CO 2017010149 A2 CO2017010149 A2 CO 2017010149A2
Authority
CO
Colombia
Prior art keywords
glut1
certain embodiments
relates
present
recombinant
Prior art date
Application number
CONC2017/0010149A
Other languages
English (en)
Inventor
Vivo Darryl De
Umrao Monani
Guangping Gao
Kristin Engelstad
Original Assignee
Univ Columbia
Univ Massachusetts
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 Univ Columbia, Univ Massachusetts filed Critical Univ Columbia
Publication of CO2017010149A2 publication Critical patent/CO2017010149A2/es

Links

Classifications

    • 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

Landscapes

  • 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 β-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.
CONC2017/0010149A 2015-03-10 2017-10-05 Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1 CO2017010149A2 (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
CO2017010149A2 true CO2017010149A2 (es) 2018-02-28

Family

ID=56879055

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2017/0010149A CO2017010149A2 (es) 2015-03-10 2017-10-05 Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión 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)

Families Citing this family (13)

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

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
NZ600121A (en) 2001-11-13 2013-12-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
EP2610265B1 (en) * 2003-05-02 2016-11-16 Centre National de la Recherche Scientifique (C.N.R.S.) Glut-1 as a receptor for HTLV envelopes and its uses
SI2292780T1 (sl) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba
ES2525143T3 (es) 2005-04-07 2014-12-18 The Trustees Of The University Of Pennsylvania Cápsides de AAVrh.64 modificado, composiciones que las contienen y usos de las mismas
WO2008134632A1 (en) * 2007-04-26 2008-11-06 President And Fellows Of Harvard College Wnt ligands involved in blood-brain barrier development and uses therefor
WO2010071832A1 (en) * 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
WO2010121010A2 (en) 2009-04-16 2010-10-21 President And Fellows Of Harvard College Methods for inhibiting starvation of a cell
CA3049237C (en) * 2010-04-23 2024-06-11 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
EP2394667A1 (en) * 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases

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
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
MX2017011615A (es) 2018-04-11
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

Similar Documents

Publication Publication Date Title
CO2017010149A2 (es) Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1
AR093355A1 (es) Composiciones y metodos para tratar proteinopatias
AR100419A1 (es) Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosómico
CL2018000587A1 (es) Adenovirus oncolítico que codifica una proteína b7 (divisional de sol. n° 2731-2017)
CO2017000506A2 (es) Receptor quimérico de antígeno cll-1
PE20160769A1 (es) Vectores del factor viii del virus adeno-asociado
NZ761655A (en) Cellular models of and therapies for ocular diseases
ECSP17059343A (es) ARNi VARIANTE
CL2018000515A1 (es) Virus de la enfermedad de newcastle y usos de los mismos (divisional sol. 2532-2015)
CO2017000510A2 (es) Constructos de car
AR091735A1 (es) Complejo de factor viii con xten y proteina factor de von willebrand (vwf) y usos del mismo
CO2018007203A2 (es) Focalización de péptidos para dirigir virus adenoasociados (aavs)
BR112017002781A2 (pt) cassete de expressão aprimorado para acondicionamento e expressão de variantes do fator viii para o tratamento de distúrbios de hemostasia
CO6470858A2 (es) Composición tensioactiva reconstituida mejorada que contiene análogos de la proteína tensioactiva b (sp-b) y de la proteína tensioactiva c (sp-c)
CL2017000647A1 (es) Proteínas de fusión, bacterias recombinantes y métodos de uso de las bacterias recombinantes
CL2020001262A1 (es) Plataforma de presentación en endosporas basadas en paenibacillus, productos y métodos relacionados.
BR112018008766A2 (pt) variantes, composições e métodos e utilizações do fator viii reduzido cpg para o tratamento de transtornos hemostásicos
EA201790378A1 (ru) Заключенное в липосому аффинное лекарственное средство
EA201791134A1 (ru) Полипептиды химерного фактора viii и их применение
BR112014005144A2 (pt) preparação e composições de enxofre com valência zero altamente biodisponíveis e seus usos
BR112018071200A2 (pt) terapia genética para o tratamento da hemofilia a
BR112014024751A2 (pt) antígenos de clostridium difficile
BR112017008962A2 (pt) proteínas de fusão inibidoras de c1 esterase e usos das mesmas
AR093569A1 (es) Tensioactivos pulmonares reconstituidos
AR113134A1 (es) Arni variante