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It was first described in <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"#jnr23755-bib-0042\"\/>, but the genetic defect for the galactocerebrosidase (GALC) gene was not discovered until the beginning of the 1970s, 20 years before the GALC cloning. Recently, in 2011, the crystal structures of the GALC enzyme and the GALC\u2010product complex were obtained. For this, compared with other LSDs, the research on possible therapeutic interventions is much more recent. Thus, it is not surprising that some treatment options are still under preclinical investigation, whereas their relevance for other pathologies of the same group has already been tested in clinical studies. This is specifically the case for pharmacological chaperone therapy (PCT), a promising strategy for selectively correcting defective protein folding and trafficking and for enhancing enzyme activity by small molecules. These compounds bind directly to a partially folded biosynthetic intermediate, stabilize the protein, and allow completion of the folding process to yield a functional protein. Here, we review the chaperones that have demonstrated potential therapeutics during preclinical studies for KD, underscoring the requirement to invigorate research for KD\u2010addressed PCT that will benefit from recent insights into the molecular understanding of GALC structure, drug design, and development in cellular models. \u00a9 2016 Wiley Periodicals, Inc.<\/jats:p>","DOI":"10.1002\/jnr.23755","type":"journal-article","created":{"date-parts":[[2016,9,17]],"date-time":"2016-09-17T11:22:31Z","timestamp":1474111351000},"page":"1220-1230","source":"Crossref","is-referenced-by-count":20,"title":["Chaperones as potential therapeutics for Krabbe disease"],"prefix":"10.1002","volume":"94","author":[{"given":"Adriana Carol Eleonora","family":"Graziano","sequence":"first","affiliation":[{"name":"Department of Biomedical and Biotechnological Science, Section of Physiology University of Catania Catania 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