FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology and is a method of treating a human patient with lysosomal acid lipase (LAL) deficiency, comprising administering to a patient an effective amount of a recombinant glycoprotein of human lysosomal acid lipase (rHGLAL), wherein the rHGLAL consists essentially of an amino acid sequence selected from a group consisting of SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4 and SEQ ID NO:19, with N-glycans associated with asparagine (Asn) residues, which correspond to Asn36, Asn101, Asn161, Asn273 or Asn321 sequence SEQ ID NO: 1, provided that the N-glycans do not contain fucose, and provided that the rGLCH does not contain O-glycans, and where the N-glycans associated with Asn residues correspond to Asn36, Asn101, Asn161, Asn273 and Asn321 sequence SEQ ID NO: 1.
EFFECT: invention provides higher clinical effectiveness in the patient suffering lysosomal acid lipase deficiency.
10 cl, 30 dwg, 6 tbl, 20 ex
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Authors
Dates
2024-12-02—Published
2020-12-22—Filed