UA128249C2 - Рекомбінантна конструкція glut1 аденоасоційованого вірусного вектора і спосіб відновлення експресії glut1 на її основі - Google Patents
Рекомбінантна конструкція glut1 аденоасоційованого вірусного вектора і спосіб відновлення експресії glut1 на її основі Download PDFInfo
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- UA128249C2 UA128249C2 UAA201709758A UAA201709758A UA128249C2 UA 128249 C2 UA128249 C2 UA 128249C2 UA A201709758 A UAA201709758 A UA A201709758A UA A201709758 A UAA201709758 A UA A201709758A UA 128249 C2 UA128249 C2 UA 128249C2
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- 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
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- 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
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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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- 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
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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
- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/63—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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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- 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)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- 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)
- Biophysics (AREA)
- Cell Biology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
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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 |
|---|---|
| UA128249C2 true UA128249C2 (uk) | 2024-05-22 |
Family
ID=56879055
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| UAA201709758A UA128249C2 (uk) | 2015-03-10 | 2016-03-10 | Рекомбінантна конструкція glut1 аденоасоційованого вірусного вектора і спосіб відновлення експресії glut1 на її основі |
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=) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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. |
| 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 |
Family Cites Families (17)
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| 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 |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The 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 |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| ES2740827T3 (es) * | 2003-05-02 | 2020-02-06 | Centre Nat Rech Scient | GLUT-1 como receptor de envolturas del HTLV y sus usos |
| JP2006345703A (ja) * | 2003-09-09 | 2006-12-28 | Osaka Industrial Promotion Organization | 高発現型汎用哺乳動物宿主細胞 |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| 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 |
| WO2010071832A1 (en) * | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| US9610363B2 (en) * | 2009-04-16 | 2017-04-04 | President And Fellows Of Harvard College | Methods for inhibiting starvation of a cell |
| EP2561073B1 (en) * | 2010-04-23 | 2016-08-24 | 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 |
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2016
- 2016-03-10 KR KR1020247024535A patent/KR20240119152A/ko not_active Ceased
- 2016-03-10 DK DK16762530.0T patent/DK3268024T3/da active
- 2016-03-10 PT PT167625300T patent/PT3268024T/pt unknown
- 2016-03-10 SG SG11201707116QA patent/SG11201707116QA/en unknown
- 2016-03-10 IL IL282053A patent/IL282053B2/en unknown
- 2016-03-10 JP JP2017548205A patent/JP2018509164A/ja active Pending
- 2016-03-10 EP EP16762530.0A patent/EP3268024B1/en active Active
- 2016-03-10 US US15/556,412 patent/US20180042991A1/en not_active Abandoned
- 2016-03-10 ES ES16762530T patent/ES2836258T3/es active Active
- 2016-03-10 KR KR1020177028554A patent/KR20180016722A/ko not_active Ceased
- 2016-03-10 AU AU2016229000A patent/AU2016229000B2/en not_active Ceased
- 2016-03-10 EA EA201791936A patent/EA036051B1/ru unknown
- 2016-03-10 MX MX2017011615A patent/MX2017011615A/es unknown
- 2016-03-10 HU HUE16762530A patent/HUE052577T2/hu unknown
- 2016-03-10 CN CN201680025266.9A patent/CN107635575A/zh active Pending
- 2016-03-10 UA UAA201709758A patent/UA128249C2/uk unknown
- 2016-03-10 WO PCT/US2016/021810 patent/WO2016145217A1/en not_active Ceased
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2017
- 2017-09-03 IL IL254286A patent/IL254286B/en active IP Right Grant
- 2017-09-08 MX MX2023001213A patent/MX2023001213A/es unknown
- 2017-09-08 CL CL2017002282A patent/CL2017002282A1/es unknown
- 2017-10-05 CO CONC2017/0010149A patent/CO2017010149A2/es unknown
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2020
- 2020-11-19 US US16/952,801 patent/US20210069292A1/en not_active Abandoned
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2021
- 2021-03-01 JP JP2021032174A patent/JP2021097689A/ja active Pending
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2023
- 2023-08-17 JP JP2023133147A patent/JP2023144087A/ja active Pending
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