STABLE EXPRESSION OF VECTORS BASED ON ADENO-ASSOCIATED VIRUS IN MINOR PATIENTS Russian patent published in 2023 - IPC A61K48/00 A61K38/37 A61K38/44 A61K38/48 C12N15/86 

FIELD: medicine.

SUBSTANCE: invention relates to the use of vectors based on an adeno-associated virus (hereinafter – AAV) for achievement of long-term expression of a transgene in the liver of a minor patient. A viral AAV particle contains capsid AAV protein of serotype 5 and AAV vector. Moreover, AAV vector contains a factor VIII transgene flanked with one or more AAV terminal inverted repeat sequences (ITR) and functionally connected to one or more regulatory transcription elements. At the same time, the factor VIII transgene encodes a functionally active factor VIII. Therapeutically effective amount of the viral AAV particle is an absolute concentration of AAV vector genome per kilogram (kg) of the body weight of an adult patient.

EFFECT: implementation of the group of inventions provides stable long-term relief of symptoms of hemophilia A in a patient after single administration of AAV-based vector to a minor patient.

60 cl, 10 dwg, 2 ex