Potential target for Krabbe disease therapy

Researchers report that the toxic glycolipid psychosine, which accumulates in people with the metabolic disorder Krabbe disease, is produced by the breakdown of galactosylceramide by the enzyme acid ceramidase (ACDase) and that removing ACDase from mouse models of Krabbe disease, either through genetic depletion or drug treatment, decreased or eliminated both psychosine production and disease symptoms.

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Article #19-12108: "Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target," by Yedda Li et al.

MEDIA CONTACT: Mark S. Sands, Washington University Medical Center, St. Louis, MO; e-mail: mssands@wustl.edu