Marc-David Ruepp, PhD & Sarah Mizielinska, PhD
King’s College, London
Dissecting the Molecular Determinants of Cytoplasmic Gain of Function Pathomechanisms in TDP - 43 - linked Amyotrophic Lateral Sclerosis
A major hallmark of ALS pathology is incorrect localisation of a protein called TDP-43. When in the incorrect location TDP-43 can initiate degeneration of brain cells. This project aims to dissect whether this toxicity depends on three different capabilities of TDP-43 by generating genetically modified stem cells which can be studied directly and used in future studies to generate mouse models.