MX2023001213A - Construcciones de vectores virales adeno-asociados de glut1 recombinantes y metodos relacionados para restablecer la expresion de glut1. - Google Patents
Construcciones de vectores virales adeno-asociados de glut1 recombinantes y metodos relacionados para restablecer la expresion de glut1.Info
- Publication number
- MX2023001213A MX2023001213A MX2023001213A MX2023001213A MX2023001213A MX 2023001213 A MX2023001213 A MX 2023001213A MX 2023001213 A MX2023001213 A MX 2023001213A MX 2023001213 A MX2023001213 A MX 2023001213A MX 2023001213 A MX2023001213 A MX 2023001213A
- Authority
- MX
- Mexico
- Prior art keywords
- glut1
- certain embodiments
- relates
- present
- recombinant
- Prior art date
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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
- 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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- 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
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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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- 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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- 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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- 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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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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- 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)
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- Engineering & Computer Science (AREA)
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- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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Abstract
La presente invención se refiere a construcciones de vectores virales adeno-asociados (rAAV) de Glut1 recombinantes y métodos relacionados para restablecer la expresión de Glut1 en mamíferos con deficiencia de Glut1. En determinadas modalidades, el rAAV además comprende un promotor de ß-actina de pollo, en donde el rAAV es capaz de cruzar la 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 modalidades, la presente invención se refiere a un kit que comprende una cubierta de un recipiente que comprende la composición descrita en la presente. En determinadas modalidades, la presente invención se refiere a métodos para restablecer el transporte de Glut1 en la BBB de un sujeto, que comprende administrarle al sujeto una cantidad eficaz de cualquiera de los vectores AAV recombinantes descritos en la presente. En determinadas modalidades, la presente invención se refiere a un método para tratar el síndrome de deficiencia de Glut1 en un sujeto que lo necesita.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562130899P | 2015-03-10 | 2015-03-10 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023001213A true MX2023001213A (es) | 2023-03-03 |
Family
ID=56879055
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2017011615A MX2017011615A (es) | 2015-03-10 | 2016-03-10 | Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1. |
| MX2023001213A MX2023001213A (es) | 2015-03-10 | 2017-09-08 | Construcciones de vectores virales adeno-asociados de glut1 recombinantes y metodos relacionados para restablecer la expresion de glut1. |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2017011615A MX2017011615A (es) | 2015-03-10 | 2016-03-10 | 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) | KR20180016722A (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 (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| HUE055862T2 (hu) | 2016-04-20 | 2021-12-28 | Centro De Investig Energeticas | Készítmények és eljárások PKLR fokozott génexpressziójára |
| 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 |
| WO2019079338A1 (en) | 2017-10-16 | 2019-04-25 | Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. | LENTIVIRAL VECTORS FOR PKLR ADMINISTRATION TO TREAT PYRUVATE KINASE DEFICIENCY |
| US12539316B2 (en) | 2018-04-11 | 2026-02-03 | Spacecraft Seven, Llc | Compositions and methods for stem cell transplant |
| RU2020134965A (ru) | 2018-04-27 | 2022-04-27 | Спейскрафт Севен, Ллк | Генная терапия при дегенерации цнс |
| KR102877876B1 (ko) * | 2018-05-08 | 2025-10-28 | 루트거스, 더 스테이트 유니버시티 오브 뉴 저지 | Aav-상용성 라미닌-링커 중합 단백질 |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| EP3856762A1 (en) * | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| SG11202110935RA (en) | 2019-05-13 | 2021-10-28 | Dna Twopointo Inc | Modifications of mammalian cells using artificial micro-rna to alter their properties and the compositions of their products |
| 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 |
| US20230248824A1 (en) * | 2020-07-09 | 2023-08-10 | The Board of Trutees of the Leland Stanford Junior University | Immune cells with increased glycolytic flux |
| JP2023536902A (ja) * | 2020-08-05 | 2023-08-30 | 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 |
| KR20230043869A (ko) | 2020-08-07 | 2023-03-31 | 스페이스크래프트 세븐, 엘엘씨 | Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법 |
| 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 |
| KR20240142487A (ko) * | 2022-02-08 | 2024-09-30 | 스페이스크래프트 세븐, 엘엘씨 | Glut1 발현을 위한 아데노-관련 바이러스 벡터 및 이의 용도 |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| CA2264482A1 (en) | 1996-09-06 | 1998-03-12 | 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 |
| JP4677187B2 (ja) | 2001-11-13 | 2011-04-27 | ザ・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 | 新規なアデノ随伴ウイルス（ａａｖ）７配列、それを含むベクターおよびそれらの使用 |
| US7642061B2 (en) * | 2003-05-02 | 2010-01-05 | Centre National De La Recherche Scientifique | 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 |
| NZ545628A (en) | 2003-09-30 | 2009-04-30 | Univ Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor |
| EP2359866B1 (en) | 2005-04-07 | 2013-07-17 | The Trustees of The University of Pennsylvania | Modified AAV rh48 capsids, compositions containing same and uses thereof |
| 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 |
| WO2010121010A2 (en) * | 2009-04-16 | 2010-10-21 | President And Fellows Of Harvard College | Methods for inhibiting starvation of a cell |
| EP3536781A1 (en) * | 2010-04-23 | 2019-09-11 | 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 |
-
2016
- 2016-03-10 KR KR1020177028554A patent/KR20180016722A/ko not_active Ceased
- 2016-03-10 KR KR1020247024535A patent/KR20240119152A/ko not_active Ceased
- 2016-03-10 WO PCT/US2016/021810 patent/WO2016145217A1/en not_active Ceased
- 2016-03-10 CN CN201680025266.9A patent/CN107635575A/zh active Pending
- 2016-03-10 SG SG11201707116QA patent/SG11201707116QA/en unknown
- 2016-03-10 UA UAA201709758A patent/UA128249C2/uk unknown
- 2016-03-10 HU HUE16762530A patent/HUE052577T2/hu unknown
- 2016-03-10 IL IL282053A patent/IL282053B2/en unknown
- 2016-03-10 EA EA201791936A patent/EA036051B1/ru unknown
- 2016-03-10 JP JP2017548205A patent/JP2018509164A/ja active Pending
- 2016-03-10 PT PT167625300T patent/PT3268024T/pt unknown
- 2016-03-10 MX MX2017011615A patent/MX2017011615A/es unknown
- 2016-03-10 US US15/556,412 patent/US20180042991A1/en not_active Abandoned
- 2016-03-10 AU AU2016229000A patent/AU2016229000B2/en not_active Ceased
- 2016-03-10 DK DK16762530.0T patent/DK3268024T3/da active
- 2016-03-10 ES ES16762530T patent/ES2836258T3/es active Active
- 2016-03-10 EP EP16762530.0A patent/EP3268024B1/en active Active
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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
-
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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