[HTML][HTML] Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated β-glucuronidase in the murine model of mucopolysaccharidosis VII
MS Sands, CA Vogler, KK Ohlemiller… - Journal of Biological …, 2001 - ASBMB
Enzyme replacement therapy (ERT) has been shown to be effective at reducing the
accumulation of undegraded substrates in lysosomal storage diseases. Most ERT studies
have been performed with recombinant proteins that are mixtures of phosphorylated and
non-phosphorylated enzyme. Because different cell types use different receptors to take up
phosphorylated or non-phosphorylated enzyme, it is difficult to determine which form of
enzyme contributed to the clinical response. Here we compare the uptake, distribution, and …
accumulation of undegraded substrates in lysosomal storage diseases. Most ERT studies
have been performed with recombinant proteins that are mixtures of phosphorylated and
non-phosphorylated enzyme. Because different cell types use different receptors to take up
phosphorylated or non-phosphorylated enzyme, it is difficult to determine which form of
enzyme contributed to the clinical response. Here we compare the uptake, distribution, and …
