FIELD: medicine.
SUBSTANCE: invention represents a recovered nucleic acid with a nucleotide sequence coding human lysosomal glucocerebrosidase protein continuously bound with C-terminal vacuolar target signal and N-terminal signal peptide of endoplasmic reticulum. The invention concerns host-cells, vectors and methods for expression and production of high-yield biologically active glucocerebrosidase (GCD) with high mannose content. The invention also concerns pharmaceutical compositions and methods of treating Gaucher disease.
EFFECT: invention allows high-efficient treatment of Gaucher disease.
54 cl, 22 dwg, 3 tbl, 7 ex
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