Title : Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target.

Pub. Date : 2019 Oct 1

PMID : 31527255






1 Functional Relationships(s)
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1 We show that pharmacological inhibition of ACDase activity with carmofur significantly decreases psychosine accumulation in cells from a Krabbe patient and prolongs the life span of the twitcher (Twi) mouse. carmofur N-acylsphingosine amidohydrolase 1 Homo sapiens