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Centre de recherche

Scientific Conference

The Conditional Knockout Approach: Cre/Lox Technology in Human Neurons

Thursday 7 November 2019 from 12:00 at 13:00


  • Christopher Patzke, PhD
    • Research associate, Südhof-lab, Stanford University School of Medicine, Department of Molecular & Cellular Physiology

The use of human pluripotent stem cells to model human diseases has become a new standard in biomedical sciences. To this end, patient-derived somatic cells are studied in vitro to mimic human pathological conditions. Particularly, the human synapse is involved in many congenital disorders, hence a systematic understanding of its function and structure is essential for the quest of therapies. In my presentation I describe as an experimental strategy the ‘Conditional Knockout Approach’ which allows to engineer disease-relevant mutations in human neurons. In combination with Cre/Lox technology, this method enables me to investigate the molecular causes of human diseases independent of the genetic background or of genetic alterations induced by clonal selection. I generated knockouts in human genes encoding for major neuronal and synaptic components including Munc18-1 (STXBP1), L1CAM, and Synapsin-1, which impact synaptic transmission, axon development, and synaptic plasticity, respectively. These tools allow me to study not only disease causing alterations but also to uncover a novel mechanism of presynaptic plasticity. Due to the ability of these cells to live for prolonged time periods, phenotypes could still be detected after 1.5 years and more.

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Updated on 11/1/2019
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