AR126841A1 - Método para obtener una cápside modificada del virus adeno-asociado - Google Patents
Método para obtener una cápside modificada del virus adeno-asociadoInfo
- Publication number
- AR126841A1 AR126841A1 ARP220102252A ARP220102252A AR126841A1 AR 126841 A1 AR126841 A1 AR 126841A1 AR P220102252 A ARP220102252 A AR P220102252A AR P220102252 A ARP220102252 A AR P220102252A AR 126841 A1 AR126841 A1 AR 126841A1
- Authority
- AR
- Argentina
- Prior art keywords
- capsid
- modified
- amino acid
- aav
- adjacent
- Prior art date
Links
- 210000000234 capsid Anatomy 0.000 title abstract 22
- 241000702421 Dependoparvovirus Species 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 3
- 235000001014 amino acid Nutrition 0.000 abstract 9
- 150000001413 amino acids Chemical class 0.000 abstract 9
- 230000003993 interaction Effects 0.000 abstract 6
- 125000000539 amino acid group Chemical group 0.000 abstract 3
- 238000002703 mutagenesis Methods 0.000 abstract 3
- 231100000350 mutagenesis Toxicity 0.000 abstract 3
- 235000018102 proteins Nutrition 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 102000004169 proteins and genes Human genes 0.000 abstract 3
- XUJNEKJLAYXESH-REOHCLBHSA-N L-Cysteine Chemical compound SC[C@H](N)C(O)=O XUJNEKJLAYXESH-REOHCLBHSA-N 0.000 abstract 2
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 abstract 2
- 235000018417 cysteine Nutrition 0.000 abstract 2
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 abstract 2
- 229930182817 methionine Natural products 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 238000006467 substitution reaction Methods 0.000 abstract 2
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 1
- 241000701945 Parvoviridae Species 0.000 abstract 1
- 241000700605 Viruses Species 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 238000004519 manufacturing process Methods 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
Classifications
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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
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
- C07K14/01—DNA viruses
- C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
-
- 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
- C12N2750/14145—Special targeting system for viral vectors
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente solicitud está relacionada con los campos de terapia génica y biología molecular. Más específicamente, la presente invención se refiere a un método para obtener una cápside modificada del virus adenoasociado (AAV) y una cápside modificada del virus adenoasociado (AAV) obtenida por el método anterior, así como a un ácido nucleico aislado que codifica la cápside modificada anterior, y a un vector basado en un virus adenoasociado recombinante para administrar a un sujeto una secuencia de ácido nucleico heterólogo que incluye la cápside modificada anterior. Reivindicación 1: Un método para producir una cápside del AAV modificada, que comprende: a) determinar los aminoácidos de la proteína del capsómero del AAV modificado, que se encuentran en la región de la interfaz de interacción de las subunidades pentaméricas adyacentes de la cápside b) alinear estructuralmente la cápside del AAV modificada con la cápside modelo para determinar la correspondencia por pares entre cada aminoácido del AAV modificado de la región de interfaz de interacción entre las subunidades pentaméricas adyacentes y el aminoácido más cercano de la cápside modelo, como análogo estructural del aminoácido inicial que se considera potencialmente sustituido, donde se entiende que la cápside molde es una cápside estructuralmente similar de un virus seleccionado de la familia Parvoviridae, que no sea AAV; c) comparar por pares los residuos de aminoácidos, determinados en la etapa b), de la cápside del AAV modificada y la cápside modelo para identificar las diferencias estructurales entre la cápside del AAV modificada y la cápside modelo en la región de la interfaz de interacción de las subunidades pentaméricas adyacentes de la cápside; d) seleccionar posiciones para la mutagénesis, en las que los residuos de aminoácidos para la mutagénesis están ubicados en la región de la interfaz de interacción de las subunidades pentaméricas adyacentes de la cápside del AAV modificada y tienen una diferencia estructural entre la cápside del AAV modificada y la cápside modelo en la región de la interfaz de interacción de las subunidades pentaméricas adyacentes de la cápside e) seleccionar un residuo de aminoácido para la mutagénesis en la posición seleccionada en la etapa d) usando uno de los siguientes principios -sustitución del aminoácido inicial en la proteína del capsómero del AAV modificado por un aminoácido de mayor volumen, distinto de la cisteína y la metionina, -sustitución del aminoácido inicial en la proteína del capsómero del AAV modificado por un aminoácido que tenga un mayor o menor número de contactos polares entre los capsómeros, distinto de la cisteína y la metionina; f) introducir una o más sustituciones de aminoácidos seleccionadas en las etapas d) - e) en la región de interfaz de interacción de las subunidades pentaméricas adyacentes de la cápside del AAV modificada.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| RU2021124731 | 2021-08-20 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR126841A1 true AR126841A1 (es) | 2023-11-22 |
Family
ID=85240917
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220102252A AR126841A1 (es) | 2021-08-20 | 2022-08-19 | Método para obtener una cápside modificada del virus adeno-asociado |
Country Status (11)
| Country | Link |
|---|---|
| EP (1) | EP4389880A1 (es) |
| CN (1) | CN118139974A (es) |
| AR (1) | AR126841A1 (es) |
| AU (1) | AU2022328531A1 (es) |
| CA (1) | CA3229580A1 (es) |
| CO (1) | CO2024001768A2 (es) |
| EC (1) | ECSP24013031A (es) |
| IL (1) | IL310949A (es) |
| MA (1) | MA64631A1 (es) |
| TW (1) | TW202317770A (es) |
| WO (1) | WO2023022631A1 (es) |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE3431140A1 (de) | 1984-08-24 | 1986-03-06 | Behringwerke Ag, 3550 Marburg | Enhancer fuer eukaryotische expressionssysteme |
| GB8717430D0 (en) | 1987-07-23 | 1987-08-26 | Celltech Ltd | Recombinant dna product |
| EP3693025B1 (en) | 2011-04-22 | 2021-10-13 | The Regents of The University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| MY172457A (en) | 2012-04-18 | 2019-11-26 | Childrens Hospital Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| CA3012711A1 (en) * | 2016-01-26 | 2017-08-03 | Defensin Therapeutics Aps | Methods for modulating intestinal microbiota |
| BR112020023393A2 (pt) * | 2018-05-16 | 2021-02-09 | Spark Therapeutics, Inc. | cassetes de expressão de a-glicosidase ácida otimizados por códon e métodos de uso dos mesmos |
-
2022
- 2022-08-18 WO PCT/RU2022/050255 patent/WO2023022631A1/ru active Application Filing
- 2022-08-18 EP EP22858841.4A patent/EP4389880A1/en active Pending
- 2022-08-18 IL IL310949A patent/IL310949A/en unknown
- 2022-08-18 CN CN202280070178.6A patent/CN118139974A/zh active Pending
- 2022-08-18 MA MA64631A patent/MA64631A1/fr unknown
- 2022-08-18 AU AU2022328531A patent/AU2022328531A1/en active Pending
- 2022-08-18 CA CA3229580A patent/CA3229580A1/en active Pending
- 2022-08-19 AR ARP220102252A patent/AR126841A1/es unknown
- 2022-08-22 TW TW111131555A patent/TW202317770A/zh unknown
-
2024
- 2024-02-19 EC ECSENADI202413031A patent/ECSP24013031A/es unknown
- 2024-02-20 CO CONC2024/0001768A patent/CO2024001768A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| ECSP24013031A (es) | 2024-03-01 |
| AU2022328531A1 (en) | 2024-03-14 |
| MA64631A1 (fr) | 2024-05-31 |
| EP4389880A1 (en) | 2024-06-26 |
| IL310949A (en) | 2024-04-01 |
| CA3229580A1 (en) | 2023-02-23 |
| CO2024001768A2 (es) | 2024-07-08 |
| TW202317770A (zh) | 2023-05-01 |
| WO2023022631A1 (ru) | 2023-02-23 |
| CN118139974A (zh) | 2024-06-04 |
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