FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology and is a method for the expression of at least one galactosidase A (α-Gal A) protein in a subject in need, including the introduction to the subject of an adeno-associated virus (AVV) expressing structure containing an apoliprotein E enhancer (APOE) associated with an alpha-1-antitrypsin (hAAT) promoter, human hemoglobin beta intron (HBB) - IGG, a signaling peptide, a cDNA transgene encoding at least one protein alpha-galactosidase (α-Gal A), and poly A signaling sequence of bovine growth hormone. The invention also concerns a pharmaceutically acceptable composition containing an AVV expressing structure for the treatment of Fabry disease.
EFFECT: invention makes it possible to effectively treat Fabry disease.
14 cl, 33 dwg, 5 tbl, 11 ex
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