TARGETED THERAPEUTIC LYSOSOMAL ENZYME FUSION PROTEINS AND USES THEREOF Russian patent published in 2019 - IPC C07K14/65 C07K14/435 C07K19/00 C12N9/24 C12N15/62 C12P21/02 A61K38/47 A61P43/00 

Abstract RU 2680581 C2

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

Aoyagi-Skarber Mika

Kristianson Tereza Margaret

Dvorak-Evell Melita

Vendt Deniel Dzh.

Long Shinong

Lebovits Dzhonatan

Gold Deniel Solomon

Dates

2019-02-22Published

2013-11-27Filed