Enhanced in vivo uptake of glucocerebrosidase
A pharmaceutical composition comprising remodelled recombinant glucocerebrosidase (GCR) is described that provides a therapeutic effect at doses that are lower then those required using remodelled naturally occurring GCR. A method of treating patients with Gaucher's disease using remodelled rec...
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Zusammenfassung: | A pharmaceutical composition comprising remodelled recombinant glucocerebrosidase (GCR) is described that provides a therapeutic effect at doses that are lower then those required using remodelled naturally occurring GCR. A method of treating patients with Gaucher's disease using remodelled recombinant GCR is also provided. In vivo uptake of exogenous molecules can be determined by extracting a mixture of cells from a subject, enriching the target cells in vitro, lysing the cells and determining the amount of exogenous molecules. |
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