Author(s): Rolfs, Prof. Arndt, MD, Eichler, Sabrina, PhD, Lukas, Jan, PhD, Cozma, Dr. rer. nat. Claudia, MD, Kramp, Guido Johannes, PhD, Kropp, Prof. Peter, MD, Böttcher, Tobias, PhD, Witt, Prof. Martin, MD, Meyer, Anja, Yang, Fan, Neßlauer, Anna-Maria
We established a long-term infusion model in mice to examine the effect of lyso-Gb1 on representative hallmark parameters of Gaucher disease. Read more!
Glucosylceramide and glucosylsphingosine are the two major storage products in Gaucher disease (GD), an inherited metabolic disorder caused by a deficiency of the lysosomal enzyme glucocerebrosidase. The pathological role of the deacylated form of glucosylceramide, glucosylsphingosine (lyso-Gb1), a recently identified sensitive and specific biomarker for GD, is not well investigated. We established a long-term infusion model in C57BL/6JRj mice to examine the effect of lyso-Gb1 on representative hallmark parameters of GD.
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