CL2017002282A1 - 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 glut1Info
- Publication number
- CL2017002282A1 CL2017002282A1 CL2017002282A CL2017002282A CL2017002282A1 CL 2017002282 A1 CL2017002282 A1 CL 2017002282A1 CL 2017002282 A CL2017002282 A CL 2017002282A CL 2017002282 A CL2017002282 A CL 2017002282A CL 2017002282 A1 CL2017002282 A1 CL 2017002282A1
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- Prior art keywords
- glut1
- present
- recombinant
- certain embodiments
- relates
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- 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
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/069—Vascular Endothelial cells
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- 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
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- 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
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- 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
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- 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
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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
-
- 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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- 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
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- 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
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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
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- 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)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
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- 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)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Biophysics (AREA)
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- Plant Pathology (AREA)
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- Toxicology (AREA)
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Abstract
<p>La presente invención se refiere a construcciones de vectores virales adeno-asociados (rAAV) de Glut1 recombinantes y métodos relacionados pare restablecer la expresión de Glut1 en mamíferos con deficiencia de Glut1 En determinadas modalidades, el rAAV además comprende un promotor de β-actina de polIo, en donde el rAAV es capaz de cruzar Ia 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 rnodalidades, Ia presente invención se refiere a un kit que comprende una cubierta de un recipiente que comprende Ia composición descrita en Ia presente. En determinadas modalidades, Ia presente invención se refiere a métodos para restablecer el transporte de GIut1 en Ia BBB de un sujeto, que comprende administrarle al sujeto una cantidad eficaz de cualquiera de los vectores AAV recombinantes descritos en Ia presente. En determinadas modalidades, la presente invención se refiere a un método para tratar el sindrome de deficiencia de Glut1 en un sujeto que lo necesita</p>
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562130899P | 2015-03-10 | 2015-03-10 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2017002282A1 true CL2017002282A1 (es) | 2018-04-13 |
Family
ID=56879055
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2017002282A CL2017002282A1 (es) | 2015-03-10 | 2017-09-08 | Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1 |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US20180042991A1 (es) |
| EP (1) | EP3268024B1 (es) |
| JP (3) | JP2018509164A (es) |
| KR (2) | KR20240119152A (es) |
| CN (1) | CN107635575A (es) |
| AU (1) | AU2016229000B2 (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 (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| 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 |
| CN109641063B (zh) | 2016-04-20 | 2022-11-15 | 能源环境和技术研究中心O.A., M.P. | 用于增强pklr的基因表达的组合物和方法 |
| 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 |
| MX2020003042A (es) * | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| 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 |
| JP7576754B2 (ja) | 2018-04-11 | 2024-11-01 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | 幹細胞移植のための組成物および方法 |
| AU2019261646A1 (en) | 2018-04-27 | 2020-11-12 | Spacecraft Seven, Llc | Gene therapy for CNS degeneration |
| IL278393B2 (en) * | 2018-05-08 | 2023-11-01 | Univ Rutgers | AAV compatible polymerization proteins bind to laminin |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| CN113383010B (zh) * | 2018-09-28 | 2025-09-09 | 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 |
| 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 |
| KR20220079857A (ko) * | 2019-09-13 | 2022-06-14 | 루트거스, 더 스테이트 유니버시티 오브 뉴 저지 | Aav-상용성 라미닌-링커 중합 단백질 |
| IL297741A (en) * | 2020-05-01 | 2022-12-01 | Atlas Biotechnology S A | Treatment and/or prevention of a disease or syndrome associated with a viral infection |
| EP4178974A4 (en) * | 2020-07-09 | 2024-10-30 | The Board of Trustees of the Leland Stanford Junior University | IMMUNE CELLS WITH INCREASED GLYCOLYTIC FLOW |
| JP7853952B2 (ja) * | 2020-08-05 | 2026-04-30 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | Ｇｌｕｔ１発現のためのアデノ随伴ウイルスベクターおよびその使用 |
| IL300263A (en) | 2020-08-07 | 2023-03-01 | Spacecraft Seven Llc | Plakophilin-2 (PKP2) gene therapy using an 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 |
| JP2025505662A (ja) * | 2022-02-08 | 2025-02-28 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | Ｇｌｕｔ１発現用アデノ随伴ウイルスベクターおよびその使用 |
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 |
| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | 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 |
| 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 |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US20100199362A1 (en) * | 2007-04-26 | 2010-08-05 | 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 |
| EP3567106A1 (en) * | 2010-04-23 | 2019-11-13 | 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 ES ES16762530T patent/ES2836258T3/es active Active
- 2016-03-10 CN CN201680025266.9A patent/CN107635575A/zh active Pending
- 2016-03-10 JP JP2017548205A patent/JP2018509164A/ja active Pending
- 2016-03-10 UA UAA201709758A patent/UA128249C2/uk unknown
- 2016-03-10 HU HUE16762530A patent/HUE052577T2/hu unknown
- 2016-03-10 KR KR1020247024535A patent/KR20240119152A/ko not_active Ceased
- 2016-03-10 EP EP16762530.0A patent/EP3268024B1/en active Active
- 2016-03-10 AU AU2016229000A patent/AU2016229000B2/en not_active Ceased
- 2016-03-10 MX MX2017011615A patent/MX2017011615A/es unknown
- 2016-03-10 EA EA201791936A patent/EA036051B1/ru unknown
- 2016-03-10 KR KR1020177028554A patent/KR20180016722A/ko not_active Ceased
- 2016-03-10 PT PT167625300T patent/PT3268024T/pt unknown
- 2016-03-10 SG SG11201707116QA patent/SG11201707116QA/en unknown
- 2016-03-10 US US15/556,412 patent/US20180042991A1/en not_active Abandoned
- 2016-03-10 IL IL282053A patent/IL282053B2/en unknown
- 2016-03-10 DK DK16762530.0T patent/DK3268024T3/da active
- 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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