Article

Recombinant NAGLU-IGF2 prevents physical and neurological disease and improves survival in Sanfilippo B syndrome

2021-08-08

Abstract excerpt

Recombinant human alpha- N -acetylglucosaminidase-insulin-like growth factor-2 (rhNAGLU-IGF2) is an investigational enzyme replacement therapy for Sanfilippo B, a lysosomal storage disease. Because recombinant human NAGLU (rhNAGLU) is poorly mannose 6-phosphorylated, we generated a fusion protein of NAGLU with IGF2 to permit its binding to the cation-independent mannose 6-phosphate receptor. We previously adminis...

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Literature Corpus work: 29a93be8-a3e9-527f-b74d-7b9f5fe16bc7
DOI: 10.1101/2021.08.06.455469

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