CN107635575A - 重组glut1腺相关病毒载体构建体以及用于恢复glut1表达的相关方法 - Google Patents

重组glut1腺相关病毒载体构建体以及用于恢复glut1表达的相关方法 Download PDF

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Publication number
CN107635575A
CN107635575A CN201680025266.9A CN201680025266A CN107635575A CN 107635575 A CN107635575 A CN 107635575A CN 201680025266 A CN201680025266 A CN 201680025266A CN 107635575 A CN107635575 A CN 107635575A
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China
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glut1
aav
sequences
raav
expression
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CN201680025266.9A
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Chinese (zh)
Inventor
D·德威伍
U·莫纳尼
G·高
K·恩格尔斯塔
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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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Publication of CN107635575A publication Critical patent/CN107635575A/zh
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    • A—HUMAN NECESSITIES
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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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
    • C12N15/11—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
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    • 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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CN201680025266.9A 2015-03-10 2016-03-10 重组glut1腺相关病毒载体构建体以及用于恢复glut1表达的相关方法 Pending CN107635575A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562130899P 2015-03-10 2015-03-10
US62/130,899 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
CN107635575A true CN107635575A (zh) 2018-01-26

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CN201680025266.9A Pending CN107635575A (zh) 2015-03-10 2016-03-10 重组glut1腺相关病毒载体构建体以及用于恢复glut1表达的相关方法

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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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CN112154209A (zh) * 2018-05-08 2020-12-29 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
CN113383010A (zh) * 2018-09-28 2021-09-10 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
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
CN116033917A (zh) * 2020-05-01 2023-04-28 阿特拉斯生物技术股份有限公司 与病毒感染相关的疾病或综合征的治疗和/或预防

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RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
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.
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
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
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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Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112154209A (zh) * 2018-05-08 2020-12-29 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
CN113383010A (zh) * 2018-09-28 2021-09-10 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
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
CN116033917A (zh) * 2020-05-01 2023-04-28 阿特拉斯生物技术股份有限公司 与病毒感染相关的疾病或综合征的治疗和/或预防

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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
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
ES2836258T3 (es) 2021-06-24
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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