Norman Kalmbach

TL;DR: It is demonstrated that three-dimensional differentiation of expandable midbrain floor plate neural progenitor cells (mfNPCs) leads to organoids that resemble key features of the human midbrain that investigate PD-relevant patho-mechanisms.

TL;DR: It is concluded that disruption of RBP homeostasis plays a critical role in FUS-ALS and can be treated by stimulating autophagy using small molecules to reduce cytoplasmic FUS, restore RBPHomeostasis and rescues motor function in vivo.

TL;DR: It is proposed that the HRE in C9ORF72 induces ALS pathogenesis via a combination of gain- and loss-of-function mechanisms.

TL;DR: A human in vitro model using skin biopsies from ChAc patients to generate disease-specific induced pluripotent stem cells (iPSCs) and an efficient differentiation protocol providing striatal medium spiny neurons is established, suggesting an actin-dependent mechanism of this dysfunctional phenotype and potential treatment targets in ChAc.

TL;DR: The data of iPSC derived MNs from familial ALS patients revealed several mutation-specific alterations in glutamate receptor properties and calcium dynamics that could play a role in ALS pathogenesis and may lead to future translational strategies with individual stratification of neuroprotective ALS treatments.