{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,26]],"date-time":"2025-10-26T21:24:39Z","timestamp":1761513879845,"version":"3.37.3"},"reference-count":37,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2016,9,17]],"date-time":"2016-09-17T00:00:00Z","timestamp":1474070400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100000065","name":"National Institute of Neurological Disorders and Stroke","doi-asserted-by":"crossref","award":["NS-36866"],"award-info":[{"award-number":["NS-36866"]}],"id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100000071","name":"National Institute of Child Health and Human Development","doi-asserted-by":"crossref","award":["HD09402"],"award-info":[{"award-number":["HD09402"]}],"id":[{"id":"10.13039\/100000071","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Children's Brain Disease Foundation."}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Neuroscience Research"],"published-print":{"date-parts":[[2016,11]]},"abstract":"<jats:p>Enzyme replacement therapy and substrate reduction therapy have proved useful in reversing many pathological consequences of many nonneural lysosomal storage diseases but have not yet reversed pathology or influenced disease outcome in Krabbe's disease (KD). This Review discusses the relative merits of stem cell therapy, molecular chaperone therapy, gene therapy, substrate reduction therapy, enzyme replacement therapy, and combination therapy. Given the limitations of these approaches, this Review introduces the idea of using tiny, 6\u2010nm, intensely fluorescent quantum dots (QDs) to deliver a cell\u2010penetrating peptide and 6 histidine residue\u2010tagged \u03b2\u2010D\u2010galactocerebrosidase across the blood\u2013brain barrier. We can therefore follow the fate of injected material and ensure that all targets are reached and that accumulated material is degraded. Uptake of lysosomal hydrolases is a complex process, and the cell\u2010penetrating peptide JB577 is uniquely able to promote endosomal egress of the QD cargo. This Review further shows that uptake may depend on the charge of the coating of the QD, specifically, that negative charge directs the cargo to neurons. Because KD involves primarily glia, specifically oligodendroglia, we experiment with many coatings and discover a coating (polyethylene glycol 600 amino) that has a positive charge and targets oligodendrocytes. A similar effect is achieved by treating with chondroitinase ABC to degrade the extracellular matrix, indicating that enzyme replacement has several hurdles to overcome before it can become a routine CNS therapy. \u00a9 2016 Wiley Periodicals, Inc.<\/jats:p>","DOI":"10.1002\/jnr.23805","type":"journal-article","created":{"date-parts":[[2016,9,17]],"date-time":"2016-09-17T11:22:31Z","timestamp":1474111351000},"page":"1293-1303","source":"Crossref","is-referenced-by-count":13,"title":["Quantum dots and potential therapy for <scp>K<\/scp>rabbe's disease"],"prefix":"10.1002","volume":"94","author":[{"given":"Glyn","family":"Dawson","sequence":"first","affiliation":[{"name":"Department of Pediatrics University of Chicago Chicago 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