Research ArticleLysosomal Storage Diseases

Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses

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Science Translational Medicine  07 Sep 2016:
Vol. 8, Issue 355, pp. 355ra118
DOI: 10.1126/scitranslmed.aad9823

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Heat shock protein to the rescue

The sphingolipidoses constitute a major subgroup of lysosomal storage diseases, a class of inherited metabolic disorders characterized by severe systemic and neurological problems. Few therapeutic options exist for treating these disorders. Kirkegaard et al. now demonstrate that increasing the expression of the molecular chaperone HSP70 through administration of either recombinant human HSP70 or the clinically tested, orally available small-molecule arimoclomol ameliorated disease manifestations, including brain pathology, in several different animal models of sphingolipidoses.