AR128450A1 - VECTORS OF RECOMBINANT ADENO-ASSOCIATED VIRUSES, AND METHODS OF USE THEREOF - Google Patents
VECTORS OF RECOMBINANT ADENO-ASSOCIATED VIRUSES, AND METHODS OF USE THEREOFInfo
- Publication number
- AR128450A1 AR128450A1 ARP230100270A ARP230100270A AR128450A1 AR 128450 A1 AR128450 A1 AR 128450A1 AR P230100270 A ARP230100270 A AR P230100270A AR P230100270 A ARP230100270 A AR P230100270A AR 128450 A1 AR128450 A1 AR 128450A1
- Authority
- AR
- Argentina
- Prior art keywords
- capsid protein
- aav
- methods
- vectors
- vector
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 3
- 239000013598 vector Substances 0.000 title abstract 2
- 241000700605 Viruses Species 0.000 title 1
- 108090000565 Capsid Proteins Proteins 0.000 abstract 6
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 6
- 239000013607 AAV vector Substances 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 239000013603 viral vector Substances 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 1
- 108700019146 Transgenes Proteins 0.000 abstract 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 238000012986 modification Methods 0.000 abstract 1
- 230000004048 modification Effects 0.000 abstract 1
- 210000000653 nervous system Anatomy 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Landscapes
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Se describen proteínas de cápside de AAV que comprenden una modificación en la secuencia de aminoácidos y vectores virales que comprenden la proteína de cápside de AAV modificada. También se describen métodos para administrar los vectores virales y cápsides virales a una célula o a un sujeto in vivo. Reivindicación 1: Un vector de virus adeno-asociado (AAV) que comprende (i) una proteína de cápside recombinante y (ii) un ácido nucleico de carga encapsulado por la proteína de cápside, en donde la proteína de cápside comprende un péptido que tiene la secuencia de cualquiera de SEQ ID Nº 12 - 20. Reivindicación 4: El vector de AAV de la reivindicación 3, en donde el transgén codifica una proteína terapéutica o ARN. Reivindicación 63: Una secuencia de ácido nucleico que codifica la proteína de cápside recombinante del vector de AAV de cualquiera de las reivindicaciones 1 a 62. Reivindicación 74: El método de la reivindicación 73, en donde el sujeto tiene una enfermedad o trastorno del sistema nervioso central.AAV capsid proteins comprising a modification in the amino acid sequence and viral vectors comprising the modified AAV capsid protein are described. Also described are methods for delivering the viral vectors and viral capsids to a cell or subject in vivo. Claim 1: An adeno-associated virus (AAV) vector comprising (i) a recombinant capsid protein and (ii) a cargo nucleic acid encapsulated by the capsid protein, wherein the capsid protein comprises a peptide having the sequence of any of SEQ ID NO: 12-20. Claim 4: The AAV vector of claim 3, wherein the transgene encodes a therapeutic protein or RNA. Claim 63: A nucleic acid sequence encoding the recombinant capsid protein of the AAV vector of any of claims 1 to 62. Claim 74: The method of claim 73, wherein the subject has a disease or disorder of the nervous system central.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063317948P | 2020-03-08 | 2020-03-08 | |
| US202063339366P | 2020-05-06 | 2020-05-06 | |
| US202263306890P | 2022-02-04 | 2022-02-04 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR128450A1 true AR128450A1 (en) | 2024-05-08 |
Family
ID=91274920
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP230100270A AR128450A1 (en) | 2020-03-08 | 2023-02-03 | VECTORS OF RECOMBINANT ADENO-ASSOCIATED VIRUSES, AND METHODS OF USE THEREOF |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR128450A1 (en) |
-
2023
- 2023-02-03 AR ARP230100270A patent/AR128450A1/en unknown
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FB | Suspension of granting procedure |