FIELD: biotechnology.
SUBSTANCE: described is a group of inventions comprising a pharmaceutical composition for treating haemophilia A, comprising a recombinant virus AAB5-FVIII-SQ in a concentration of about 1E12 vg/ml to about 2E14 vg/ml, method of treating a subject suffering from hemophilia A, a method for reducing bleeding time in a haemorrhage episode in a subject suffering from haemophilia A, a method for increasing expression of factor VIII protein in a subject, method of treating a subject suffering from hemophilia A, comprising steps of determining the absence of antibodies to the AACB capsid in the serum of said subject and administering the above pharmaceutical composition, and a method of treating a subject suffering from hemophilia A, comprising the steps of administering to said subject a pharmaceutical composition and, after administering said pharmaceutical composition, determining the absence or presence of the antibodies to the AACB capsid in the serum of said subject.
EFFECT: invention extends the range of products for treating haemophilia A.
26 cl, 49 dwg, 1 tbl, 8 ex
Title | Year | Author | Number |
---|---|---|---|
VECTORS OF EXPRESSION OF VIII FACTOR BASED ON ADENO-ASSOCIATED VIRUS | 2014 |
|
RU2710307C2 |
FACTOR VIII EXPRESSION VECTORS BASED ON AN ADENO-ASSOCIATED VIRUS | 2014 |
|
RU2777372C2 |
NON-VIRAL DNA VECTORS AND OPTIONS FOR THEIR USE FOR EXPRESSION OF THERAPEUTIC AGENT BASED ON PHENYLALANINE HYDROXYLASE (PAH) | 2020 |
|
RU2814137C2 |
NON-VIRAL DNA VECTORS AND OPTIONS FOR THEIR USE FOR EXPRESSION OF THERAPEUTIC AGENT BASED ON FACTOR VIII (FVIII) | 2020 |
|
RU2812852C2 |
NON-VIRAL DNA VECTORS AND THEIR USE FOR THE PRODUCTION OF ANTIBODIES AND FUSION PROTEINS | 2019 |
|
RU2800914C2 |
CLOSED-ENDED DNA VECTORS OBTAINED BY CELL-FREE SYNTHESIS, AND METHOD OF PRODUCING CEDNA VECTORS | 2019 |
|
RU2820586C2 |
STABLE EXPRESSION OF VECTORS BASED ON ADENO-ASSOCIATED VIRUS IN MINOR PATIENTS | 2019 |
|
RU2792432C2 |
CONTROLLED EXPRESSION OF TRANSGENES USING CLOSED-END DNA VECTORS (ceDNA) | 2019 |
|
RU2816871C2 |
METHODS FOR TREATMENT OF PHENYLKETONURIA | 2019 |
|
RU2788484C2 |
GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A | 2017 |
|
RU2762257C2 |
Authors
Dates
2021-02-05—Published
2016-09-23—Filed