Institute of Stem Cell Research

The institute is devoted to understand stem cell function better – especially how they generate the functional cells of an organ. This implies studying the balance between self-renewal and differentiation and generally how cell fate is established and stabilized. We use molecular tools, in particular CRISPR and viral vector tools, as well as cell biological tools to understand and manipulate these fundamental processes in order to utilize the mechanisms identified for replacing cells lost in disease. We have implemented this by the discovery of glia-to-neuron reprogramming (Heins et al., Nature Neuroscience, 2002), which we have by now established not only in the murine injured brain in vivo (Gascón et al., Cell Stem Cell, 2016), but also for human glial cells that we can efficiently convert into new neurons (Sonsalla et al., Neuron, 2024). Unexpectedly, not only transcriptional regulation plays a key role in direct reprogramming (Pereira et al., Nature Neuroscience, 2024), but also the highly cell-type-specific composition of organelles, such as mitochondria (Russo et al., Cell Stem Cell, 2021). Indeed, we discovered recently that pan-cellular organelles considered to function rather similar across cell types differ profoundly between even closely-related cell types (Camargo et al., Nature, 2019; O’Neill et al., Science, 2022). This discovery of organellar heterogeneity (Schieweck and Götz, Genes Dev., 2024) allows not only to understand how ubiquitous proteins can cause an organ-specific phenotype when mutated in patients (because only in some organs they are at the centrosome and cause a specific phenotype there), but also to improve fate conversion towards more healthy neurons (Russo et al., Cell Stem Cell, 2021; Sonsalla et al., Neuron, 2024).

Principle investigator Dr. Stefan H. Stricker joined the Institute for Stem Cell Research at the Helmholtz Zentrum München as a guest scientist associated with the Ludwig-Maximilians-Universität Munich. Since December 2016 he is the head of the MCN Junior Research group "Epigenome Editing" at the BioMedical Center.

We investigate the mechanisms and metabolic pathways driving the direct reprogramming of differentiated mouse and human cells into functional neurons, aiming at a comprehensive understanding of the conversion process.

The Gene Editing Group is dedicated to discovering, developing, and applying innovative genetic tools that drive translational advances in cell and gene therapies.