Capturing the biology of disease severity in an organoid model of LIS1-lissencephaly
Lea Zillich, Matteo Gasparotto, Andrea Carlo Rossetti, Olivia Fechtner, Camille Maillard, Anne Hoffrichter, Eric Zillich, Ammar Jabali, Fabio Marsoner, Annasara Artioli, Ruven Wilkens, Christina B. Schroeter, Andreas Hentschel, Stephanie H. Witt, Nico Melzer, , Julia Ladewig
This study uses LIS1-lissencephaly patient organoids to identify severity-dependent phenotypes and molecular mechanisms, and demonstrates that mTOR inhibitors can rescue disease-related changes.
bioRxiv · Dec 19, 2022 · PreprintNeuroscience