DK3268024T3 - Rekombinante glut1-adeno-associerede virusvektorkonstrukter og relaterede fremgangsmåder til genoprettelse af glut1-ekspression - Google Patents

Rekombinante glut1-adeno-associerede virusvektorkonstrukter og relaterede fremgangsmåder til genoprettelse af glut1-ekspression Download PDF

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Publication number
DK3268024T3
DK3268024T3 DK16762530.0T DK16762530T DK3268024T3 DK 3268024 T3 DK3268024 T3 DK 3268024T3 DK 16762530 T DK16762530 T DK 16762530T DK 3268024 T3 DK3268024 T3 DK 3268024T3
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glut1
recovering
associated virus
recombinant
related methods
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DK16762530.0T
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English (en)
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Vivo Darryl De
Umrao Monani
Guangping Gao
Kristin Engelstad
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Univ Columbia
Univ Massachusetts
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    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
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  • Virology (AREA)
  • Toxicology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
DK16762530.0T 2015-03-10 2016-03-10 Rekombinante glut1-adeno-associerede virusvektorkonstrukter og relaterede fremgangsmåder til genoprettelse af glut1-ekspression DK3268024T3 (da)

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
DK3268024T3 true DK3268024T3 (da) 2020-12-07

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DK16762530.0T DK3268024T3 (da) 2015-03-10 2016-03-10 Rekombinante glut1-adeno-associerede virusvektorkonstrukter og relaterede fremgangsmåder til genoprettelse af glut1-ekspression

Country Status (19)

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

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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
EP3790394A4 (en) * 2018-05-08 2022-07-27 Rutgers, the State University of New Jersey POLYMERIZING PROTEINS AAV-COMPATIBLE LAMININ LINKER
KR20210068068A (ko) * 2018-09-28 2021-06-08 보이저 테라퓨틱스, 인크. 조작된 프로모터를 갖는 프라탁신 발현 구축물 및 그의 사용 방법
JP7284833B2 (ja) 2019-05-13 2023-05-31 ディーエヌエー ツーポイントオー インク. 特性を変えるための人工マイクロrnaを用いた哺乳類細胞の改変及びその生産物の組成物
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
CA3174939A1 (en) * 2020-05-01 2021-11-04 Nikolay ZHUKOVSKY Treatment and/or prevention of a disease or a syndrome related to a virus infection
CA3185234A1 (en) * 2020-07-09 2022-01-13 Dorota Danuta KLYSZ Immune cells with increased glycolytic flux
AU2021321412A1 (en) * 2020-08-05 2023-04-06 Spacecraft Seven, Llc Adeno-associated viral vector for GLUT1 expression and uses thereof
AU2021320902A1 (en) 2020-08-07 2023-04-06 Spacecraft Seven, Llc Plakophilin-2 (PKP2) gene therapy using AAV vector
WO2022051627A2 (en) * 2020-09-04 2022-03-10 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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US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
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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
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NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
WO2004096841A1 (en) * 2003-05-02 2004-11-11 Centre National De La Recherche Scientifique Glut-1 as a receptor for htlv envelopes and its uses
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
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
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Also Published As

Publication number Publication date
CN107635575A (zh) 2018-01-26
KR20180016722A (ko) 2018-02-19
EA201791936A1 (ru) 2018-01-31
EP3268024A1 (en) 2018-01-17
CO2017010149A2 (es) 2018-02-28
AU2016229000B2 (en) 2021-11-04
IL254286A0 (en) 2017-10-31
PT3268024T (pt) 2020-12-09
HUE052577T2 (hu) 2021-05-28
JP2021097689A (ja) 2021-07-01
IL282053B2 (en) 2023-03-01
AU2016229000A1 (en) 2017-09-21
JP2023144087A (ja) 2023-10-06
CL2017002282A1 (es) 2018-04-13
IL254286B (en) 2021-04-29
EA036051B1 (ru) 2020-09-18
WO2016145217A1 (en) 2016-09-15
EA201791936A8 (ru) 2018-11-30
SG11201707116QA (en) 2017-09-28
US20180042991A1 (en) 2018-02-15
EP3268024A4 (en) 2018-08-01
MX2017011615A (es) 2018-04-11
BR112017019294A2 (pt) 2018-05-02
CA2978917A1 (en) 2016-09-15
IL282053B (en) 2022-11-01
IL282053A (en) 2021-05-31
EP3268024B1 (en) 2020-09-09
US20210069292A1 (en) 2021-03-11
ES2836258T3 (es) 2021-06-24
JP2018509164A (ja) 2018-04-05
MX2023001213A (es) 2023-03-03

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