FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology, specifically to therapeutic expression vectors; it can be used in medicine for the treatment of Pompe disease (GSDII) and Cori disease (GSDIII). A recombinant vector based on an adeno-associated virus (AAV) is proposed for the expression of truncated polypeptide of alpha-glycosidase (hereinafter – GAA).
EFFECT: invention provides for expression and secretion of human GAA variants at higher levels compared to human GAA protein of a wild type, which provides improved correction of pathological accumulation of glycogen in the entire body and induction of immunological tolerance to GAA.
17 cl, 1 tbl, 10 dwg, 1 ex
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