Therapeutic delivery of proteins to macrophages: implications for treatment of Gaucher's disease
Mistry P, Wraight E, Cox T. Therapeutic delivery of proteins to macrophages: implications for treatment of Gaucher's disease. The Lancet 1996, 348: 1555-1559. PMID: 8950883, DOI: 10.1016/s0140-6736(96)04451-0.Peer-Reviewed Original ResearchConceptsGaucher diseaseBone marrowType 1 Gaucher's diseaseActivity of glucocerebrosidaseReceptor-mediated uptakeAcid beta-glucosidase activityAnalysis of bloodAvid uptakeBolus injectionHigh doseGaucher cellsHealthy individualsTracer enzymeGamma scintigraphyReceptor activityMacrophage systemDiseaseLysosomal disordersPlacental enzymeTissue distributionHigh-affinity targetingSaturable uptakePrimary defectScintigraphyVivo