Quantum dots and potential therapy for Krabbe's disease.

摘要: Enzyme replacement therapy and substrate reduction have proved useful in reversing many pathological consequences of nonneural lysosomal storage diseases but not yet reversed pathology or influenced disease outcome Krabbe's (KD). This Review discusses the relative merits stem cell therapy, molecular chaperone gene enzyme combination therapy. Given limitations these approaches, this introduces idea using tiny, 6-nm, intensely fluorescent quantum dots (QDs) to deliver a cell-penetrating peptide 6 histidine residue-tagged β-D-galactocerebrosidase across blood-brain barrier. We can therefore follow fate injected material ensure that all targets are reached accumulated is degraded. Uptake hydrolases complex process, JB577 uniquely able promote endosomal egress QD cargo. further shows uptake may depend on charge coating QD, specifically, negative directs cargo neurons. Because KD involves primarily glia, specifically oligodendroglia, we experiment with coatings discover (polyethylene glycol 600 amino) has positive oligodendrocytes. A similar effect achieved by treating chondroitinase ABC degrade extracellular matrix, indicating several hurdles overcome before it become routine CNS © 2016 Wiley Periodicals, Inc.