Neurological and psychiatric disorders present a staggering health-care burden, costing around 800 billion euros per year in the EU and affecting almost 180 million people. Currently, development of new therapies for these disorders is quite unsuccessful. Most therapy design relies on heterologous cellular models (mitotic cells). However, models that model the human brain (post-mitotic neurons) more closely are still scarce. At the Functional Genomics department of Clinical Genetics, Amsterdam UMC, and the VU University, Claudia Persoon, Ruud Toonen and Matthijs Verhage developed new preclinical screening assays for therapy design, based on functional, mature human neurons and CRISPR-Cas9 technology. Human neurons, derived from patient skin cells, mimic in a tissue culture setting the situation in the patients’ brain (‘patient-brain-in-a-dish’). High-end technology is established, such as live cell imaging and electrophysiology, to assess efficacy of novel therapies. Together these assays will greatly enhance identification of successful treatment strategies.
Claudia Persoon obtained this personal NWO take-off grant to investigate the commercial potential of these new preclinical human assays. She will reach out to and collaborating with the pharmaceutical industry through a spin-off company from the VU university.