FIELD: biotechnology.
SUBSTANCE: present invention relates to the field of biotechnology, specifically to options of acid α-glucosidase (hereinafter – GAA); it can be used in medicine for the treatment of Pompe’s disease (glycogen storage disease type II; hereinafter – GSDII) and Cori’s disease (hereinafter – GSDIII). Chimeric polypeptide GAA contains a fragment of signal peptide with SEQ ID NO: 2, 3, or 4 and a functional fragment of human GAA at least 90% identical to a sequence SEQ ID NO: 5, or 36.
EFFECT: invention provides for the expression and secretion of human GAA options at higher levels compared to human GAA protein of a wild type, which provides improved correction of pathological glycogen accumulation in the entire organism and induction of immunological tolerance to GAA.
9 cl, 4 tbl, 11 dwg, 1 ex
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