FIELD: biotechnology; genetic engineering.
SUBSTANCE: recovered AAV capsid protein contains a sequence selected from the group consisting of: SEQ ID NO: from 51 to 61. Recombinant expression vector contains a nucleic acid sequence coding the isolated capsid AAV protein. Composition for transgene delivery to the patient contains isolated capsid AAV protein and a pharmaceutically acceptable carrier. Recombinant AAV (rAAV) contains extracted capsid AAV protein. Composition for transgene delivery to a patient comprises recombinant rAAV and a pharmaceutically acceptable carrier. Method for delivering a transgene to a patient comprises administering rAAV to a patient, wherein rAAV contains at least one transgene and in which rAAV infects target tissue cells. Method for creating a somatic transgenic animal model involves administering recombinant rAAV to an animal other than a human, wherein rAAV contains at least one transgene and in which rAAV infects a cell of a target tissue of an animal other than a human. Kit for producing rAAV contains a container containing recombinant AAV having an isolated capsid AAV protein.
EFFECT: group of inventions provides improved properties of tissue tropism.
16 cl, 4 dwg, 5 tbl, 1 ex
Title | Year | Author | Number |
---|---|---|---|
CLOSED-END LINEAR DUPLEX DNA FOR NON-VIRAL GENE TRANSFER | 2017 |
|
RU2752882C2 |
AAV-BASED HUNTINGTON'S DISEASE TREATMENT | 2017 |
|
RU2749971C2 |
ADENO-ASSOCIATED VIRUS VECTOR | 2015 |
|
RU2743382C2 |
RNAi AGENT OPTION | 2018 |
|
RU2789647C2 |
ADENO-ASSOCIATED VIRAL VECTORS FOR TREATMENT OF MYOCILIN (MYOC) GLAUCOMA | 2015 |
|
RU2746991C2 |
ADENO-ASSOCIATED VIRUS VECTORS FOR TREATING MYOCILIN (MYOC) GLAUCOMA | 2015 |
|
RU2718047C2 |
MODIFIED FREEDREICH ATAXY GENES AND VECTORS FOR GENE THERAPY | 2016 |
|
RU2743792C2 |
MODIFIED CAPSID PROTEINS FOR IMPROVED DELIVERY OF PARVOVIRUS VECTORS | 2016 |
|
RU2751953C2 |
ISOLATED MODIFIED CAPSID PROTEIN VPI OF ADENO-ASSOCIATED VIRUS SEROTYPE 5 (AAV5), CAPSID AND VECTOR BASED THEREON | 2021 |
|
RU2820088C1 |
COMPLETELY SCALABLE METHOD FOR PRODUCING rAAV BASED ON COLUMNS | 2017 |
|
RU2754467C2 |
Authors
Dates
2020-12-14—Published
2015-10-21—Filed