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Maik Liedtke

Untersuchungen über den Einfluss eines gestörten Organellentransports auf die Autophagie und Mitophagie im Morbus Niemann Pick Typ C, unter Verwendung eines humanen Zellmodells aus Patienten-spezifischen neural differenzierten induzierten pluripotenten Stammzellen

Universität Rostock, 2022

Abstract: Niemann-Pick type C (NP-C) disease is a neurovisceral lysosomal storage disorder. In neurally differentiated iPSCs, disturbances in the transport of mitochondria and lysosomes were detected. In addition, degradation of mitochondria via mitophagy, as well as other material via autophagy, was reduced, which was due to problems in the induction of these processes. Thus, improved recognition of material to be degraded, may offer a promising new avenue of intervention to positively impact the pathophysiology of NP-C.

doctoral thesis   free access    


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