The role of impaired de novo coenzyme. A biosynthesis in pantothenate-kinase associated neurodegeneration: insight from a Drosophila model

PhD ceremony: Mr. A. Rana, 16.15 uur, Academiegebouw, Broerstraat 5, Groningen

Title: The role of impaired de novo coenzyme. A biosynthesis in pantothenate-kinase associated neurodegeneration: insight from a Drosophila model

Promotor(s): prof. O.C.M. Sibon, prof. H.H. Kampinga

Faculty: Medical Sciences

In this thesis, we characterize the fly model for PKAN (Pantothenate Kinase-Associated Neurodegeneration) and show that model fly have shorter life and brain degeneration similar to PKAN patients. We also show that adding pantethine (a naturally occurring substance) to the food of the sick fruit flies with PKAN resulted in an increased life span, increased ability to climb and that the brains of the ‘sick’ flies looked better. Pantethine also works in a PKAN model of human cells. Finally, we show that wild-type mice could tolerate high doses of pantethine. Our results indicate that pantethine can be further developed as a potential therapy for the PKAN disease.