ES2836258T3 - Construcciones de vectores virales adenoasociados de GLUT1 recombinante y métodos relacionados para restaurar la expresión de GLUT1 - Google Patents

Construcciones de vectores virales adenoasociados de GLUT1 recombinante y métodos relacionados para restaurar la expresión de GLUT1 Download PDF

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ES2836258T3
ES2836258T3 ES16762530T ES16762530T ES2836258T3 ES 2836258 T3 ES2836258 T3 ES 2836258T3 ES 16762530 T ES16762530 T ES 16762530T ES 16762530 T ES16762530 T ES 16762530T ES 2836258 T3 ES2836258 T3 ES 2836258T3
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glut1
sequences
expression
raav
recombinant
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Spanish (es)
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Vivo Darryl De
Umrao Monani
Guangping Gao
Kristin Engelstad
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Columbia University in the City of New York
University of Massachusetts Amherst
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Columbia University in the City of New York
University of Massachusetts Amherst
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    • C12N5/0602Vertebrate cells
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
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    • AHUMAN NECESSITIES
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    • 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
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    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • 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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    • 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
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    • A61P25/28Drugs 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
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • 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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    • C12N15/09Recombinant 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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    • 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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  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
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ES16762530T 2015-03-10 2016-03-10 Construcciones de vectores virales adenoasociados de GLUT1 recombinante y métodos relacionados para restaurar la expresión de GLUT1 Active ES2836258T3 (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
ES2836258T3 true ES2836258T3 (es) 2021-06-24

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ES16762530T Active ES2836258T3 (es) 2015-03-10 2016-03-10 Construcciones de vectores virales adenoasociados de GLUT1 recombinante y métodos relacionados para restaurar la expresión de GLUT1

Country Status (18)

Country Link
US (2) US20180042991A1 (https=)
EP (1) EP3268024B1 (https=)
JP (3) JP2018509164A (https=)
KR (2) KR20240119152A (https=)
CN (1) CN107635575A (https=)
AU (1) AU2016229000B2 (https=)
CL (1) CL2017002282A1 (https=)
CO (1) CO2017010149A2 (https=)
DK (1) DK3268024T3 (https=)
EA (1) EA036051B1 (https=)
ES (1) ES2836258T3 (https=)
HU (1) HUE052577T2 (https=)
IL (2) IL282053B2 (https=)
MX (2) MX2017011615A (https=)
PT (1) PT3268024T (https=)
SG (1) SG11201707116QA (https=)
UA (1) UA128249C2 (https=)
WO (1) WO2016145217A1 (https=)

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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
HUE055862T2 (hu) 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
AU2018338728B2 (en) * 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
US12441998B2 (en) 2017-10-12 2025-10-14 The Trustees Of Columbia University In The City Of New York SLC2A1 lncRNA as a biologic and related treatments and methods
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.
AU2019265663B2 (en) * 2018-05-08 2024-06-13 Rutgers, The State University Of New Jersey AAV-compatible laminin-linker polymerization proteins
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US11162102B2 (en) 2019-05-13 2021-11-02 Dna Twopointo Inc. Modifications of mammalian cells using artificial micro-RNA to alter their properties and the compositions of their products
AU2020346062A1 (en) * 2019-09-13 2022-03-24 Rutgers, The State University Of New Jersey AAV-compatible laminin-linker polymerization proteins
US20230302104A1 (en) * 2020-05-01 2023-09-28 Atlas Biotechnology S.A. 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
CN116113700A (zh) * 2020-08-05 2023-05-12 太空飞船七有限责任公司 用于glut1表达的腺相关病毒载体及其用途
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
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
IL314602A (en) * 2022-02-08 2024-09-01 Spacecraft Seven Llc Adeno-associated viral vector for GLUT1 expression and uses thereof

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Also Published As

Publication number Publication date
JP2021097689A (ja) 2021-07-01
NZ735046A (en) 2024-11-29
JP2023144087A (ja) 2023-10-06
CA2978917A1 (en) 2016-09-15
KR20240119152A (ko) 2024-08-06
HUE052577T2 (hu) 2021-05-28
MX2017011615A (es) 2018-04-11
SG11201707116QA (en) 2017-09-28
JP2018509164A (ja) 2018-04-05
CL2017002282A1 (es) 2018-04-13
US20210069292A1 (en) 2021-03-11
BR112017019294A2 (pt) 2018-05-02
EP3268024B1 (en) 2020-09-09
UA128249C2 (uk) 2024-05-22
CN107635575A (zh) 2018-01-26
AU2016229000A1 (en) 2017-09-21
EP3268024A4 (en) 2018-08-01
IL254286A0 (en) 2017-10-31
CO2017010149A2 (es) 2018-02-28
WO2016145217A1 (en) 2016-09-15
AU2016229000B2 (en) 2021-11-04
PT3268024T (pt) 2020-12-09
IL282053B (en) 2022-11-01
EA201791936A1 (ru) 2018-01-31
IL282053B2 (en) 2023-03-01
IL282053A (en) 2021-05-31
IL254286B (en) 2021-04-29
DK3268024T3 (da) 2020-12-07
MX2023001213A (es) 2023-03-03
US20180042991A1 (en) 2018-02-15
KR20180016722A (ko) 2018-02-19
EA201791936A8 (ru) 2018-11-30
EP3268024A1 (en) 2018-01-17
EA036051B1 (ru) 2020-09-18

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