FIELD: biotechnology.
SUBSTANCE: present invention relates to the field of biotechnology, specifically to therapeutic fusion proteins that can be used in medicine to treat type IIIB mucopolysaccharidosis (Sanfillipo B Syndrome). Said fusion protein contains (a) human alpha-N-acetylglucosaminidase (Naglu), (b) IGF-II mutein with a mutation that eliminates at least one protease cleavage site by furin in said IGF-II mutein, and (c) a spacer peptide between said lysosomal enzyme and said IGF-II mutein. Said spacer peptide contains an amino acid sequence selected from GGGGSGGGGSGGGGSGGGGSGGGPS, GAPGGSPAGSPTSTEEGTSESATPESGPGTSTEPSEGSAPGSPAGSPTSTGPSGAP, GAPGGGSPAPAPTPAPAPTPAPAGGGPSGAP, GAPGGGSPAEAAAKEAAAKEAAAKEAAAKEAAAKAPSGGGGAP and GGGGAGGGGAGGGGAGGGGAGGGPS.
EFFECT: invention effectively reduces the levels of glycosaminoglycans during treatment of type IIIB mucopolysaccharidosis (Sanfillipo B Syndrome).
20 cl, 3 dwg, 2 tbl, 6 ex
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Authors
Dates
2019-02-22—Published
2013-11-27—Filed