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
Links
- 239000013603 viral vector Substances 0.000 title abstract 2
- 101100393884 Drosophila melanogaster Glut1 gene Proteins 0.000 abstract 4
- 101150058068 SLC2A1 gene Proteins 0.000 abstract 4
- 230000008499 blood brain barrier function Effects 0.000 abstract 3
- 210000001218 blood-brain barrier Anatomy 0.000 abstract 3
- 239000013607 AAV vector Substances 0.000 abstract 1
- 208000007686 GLUT1 deficiency syndrome Diseases 0.000 abstract 1
- 241000287828 Gallus gallus Species 0.000 abstract 1
- 108700006771 Glut1 Deficiency Syndrome Proteins 0.000 abstract 1
- 241000124008 Mammalia Species 0.000 abstract 1
- 230000002950 deficient Effects 0.000 abstract 1
Classifications
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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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
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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
-
- 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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- 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
- 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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- 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
- 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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Cell Biology (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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 (19)
| Country | Link |
|---|---|
| US (2) | US20180042991A1 (es) |
| EP (1) | EP3268024B1 (es) |
| JP (3) | JP2018509164A (es) |
| KR (1) | KR20180016722A (es) |
| CN (1) | CN107635575A (es) |
| AU (1) | AU2016229000B2 (es) |
| BR (1) | BR112017019294A2 (es) |
| CA (1) | CA2978917A1 (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) |
| WO (1) | WO2016145217A1 (es) |
Families Citing this family (13)
| 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 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| EP3790394A4 (en) * | 2018-05-08 | 2022-07-27 | Rutgers, the State University of New Jersey | POLYMERIZING PROTEINS AAV-COMPATIBLE LAMININ LINKER |
| KR20210068068A (ko) * | 2018-09-28 | 2021-06-08 | 보이저 테라퓨틱스, 인크. | 조작된 프로모터를 갖는 프라탁신 발현 구축물 및 그의 사용 방법 |
| JP7284833B2 (ja) | 2019-05-13 | 2023-05-31 | ディーエヌエー ツーポイントオー インク． | 特性を変えるための人工マイクロｒｎａを用いた哺乳類細胞の改変及びその生産物の組成物 |
| 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 |
| CA3185234A1 (en) * | 2020-07-09 | 2022-01-13 | Dorota Danuta KLYSZ | Immune cells with increased glycolytic flux |
| AU2021321412A1 (en) * | 2020-08-05 | 2023-04-06 | Spacecraft Seven, Llc | Adeno-associated viral vector for GLUT1 expression and uses thereof |
| AU2021320902A1 (en) | 2020-08-07 | 2023-04-06 | Spacecraft Seven, Llc | Plakophilin-2 (PKP2) gene therapy using AAV vector |
| 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 |
| WO2023154763A2 (en) * | 2022-02-08 | 2023-08-17 | Spacecraft Seven, Llc | Adeno-associated viral vector for glut1 expression and uses thereof |
Family Cites Families (16)
| 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 |
| WO1998010088A1 (en) | 1996-09-06 | 1998-03-12 | 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 |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| WO2004096841A1 (en) * | 2003-05-02 | 2004-11-11 | Centre National De La Recherche Scientifique | Glut-1 as a receptor for htlv envelopes and its uses |
| ES2648241T3 (es) | 2003-09-30 | 2017-12-29 | The Trustees Of The University Of Pennsylvania | Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos |
| 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 |
| DK2826860T3 (en) * | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
| 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 CA CA2978917A patent/CA2978917A1/en active Pending
- 2016-03-10 AU AU2016229000A patent/AU2016229000B2/en active Active
- 2016-03-10 SG SG11201707116QA patent/SG11201707116QA/en unknown
- 2016-03-10 EP EP16762530.0A patent/EP3268024B1/en active Active
- 2016-03-10 EA EA201791936A patent/EA036051B1/ru unknown
- 2016-03-10 US US15/556,412 patent/US20180042991A1/en not_active Abandoned
- 2016-03-10 CN CN201680025266.9A patent/CN107635575A/zh active Pending
- 2016-03-10 JP JP2017548205A patent/JP2018509164A/ja active Pending
- 2016-03-10 HU HUE16762530A patent/HUE052577T2/hu unknown
- 2016-03-10 PT PT167625300T patent/PT3268024T/pt unknown
- 2016-03-10 DK DK16762530.0T patent/DK3268024T3/da active
- 2016-03-10 WO PCT/US2016/021810 patent/WO2016145217A1/en active Application Filing
- 2016-03-10 ES ES16762530T patent/ES2836258T3/es active Active
- 2016-03-10 MX MX2017011615A patent/MX2017011615A/es unknown
- 2016-03-10 IL IL282053A patent/IL282053B2/en unknown
- 2016-03-10 KR KR1020177028554A patent/KR20180016722A/ko not_active Application Discontinuation
- 2016-03-10 BR BR112017019294A patent/BR112017019294A2/pt active Search and Examination
-
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 active Pending
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2021
- 2021-03-01 JP JP2021032174A patent/JP2021097689A/ja active Pending
-
2023
- 2023-08-17 JP JP2023133147A patent/JP2023144087A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CN107635575A (zh) | 2018-01-26 |
| DK3268024T3 (da) | 2020-12-07 |
| KR20180016722A (ko) | 2018-02-19 |
| EA201791936A1 (ru) | 2018-01-31 |
| EP3268024A1 (en) | 2018-01-17 |
| CO2017010149A2 (es) | 2018-02-28 |
| AU2016229000B2 (en) | 2021-11-04 |
| IL254286A0 (en) | 2017-10-31 |
| PT3268024T (pt) | 2020-12-09 |
| HUE052577T2 (hu) | 2021-05-28 |
| JP2021097689A (ja) | 2021-07-01 |
| IL282053B2 (en) | 2023-03-01 |
| AU2016229000A1 (en) | 2017-09-21 |
| JP2023144087A (ja) | 2023-10-06 |
| CL2017002282A1 (es) | 2018-04-13 |
| IL254286B (en) | 2021-04-29 |
| EA036051B1 (ru) | 2020-09-18 |
| WO2016145217A1 (en) | 2016-09-15 |
| EA201791936A8 (ru) | 2018-11-30 |
| SG11201707116QA (en) | 2017-09-28 |
| US20180042991A1 (en) | 2018-02-15 |
| EP3268024A4 (en) | 2018-08-01 |
| MX2017011615A (es) | 2018-04-11 |
| BR112017019294A2 (pt) | 2018-05-02 |
| CA2978917A1 (en) | 2016-09-15 |
| IL282053B (en) | 2022-11-01 |
| IL282053A (en) | 2021-05-31 |
| EP3268024B1 (en) | 2020-09-09 |
| US20210069292A1 (en) | 2021-03-11 |
| ES2836258T3 (es) | 2021-06-24 |
| JP2018509164A (ja) | 2018-04-05 |
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