iPSC-derived neural cells for drug discovery of mitochondrial disorders

Mutations in mitochondrial DNA (mtDNA) cause diseases typically affecting the nervous system and for which no effective treatment exists. It has been difficult to develop animal models of mitochondrial diseases due to challenges of engineering mtDNA. Existing cellular models often lack the metabolic features of neural cells and do not provide the patient-specific match between mitochondrial and nuclear genomes. 

Here we show that neural cells derived from human induced pluripotent stem cells (iPSCs) display the correct functional and bioenergetics properties to investigate the neurological impairment associated with mitochondrial disorders. We used patient iPSC-derived neural cells carrying mutations in the mitochondrial gene MT-ATP6 to carry out a proof-of-principle high-throughput compound screening to identify potential novel treatments.

In this webinar we will discuss:

  • What is the bioenergetic profile of human iPSC-derived neural cells (including neural progenitors, neurons and glia)
  • How mtDNA mutations affect neural cells in terms of bioenergetic and mitochondrial calcium homeostasis
  • How to use iPSC-derived neural cells to set up compound screening approaches

For Research Use Only.  Not for use in diagnostic procedures.

Thursday, October 26, 2017
7:00 PDT (Los Angeles)
10:00 EDT (New York)
16:00 CEST (Berlin)

Available On Demand after October 26 

All registrants will be notified when available 

Presented by:

Alessandro Prigione, M.D, Ph.D.
Independent Team Leader, Max Delbrueck Center for Molecular Medicine (MDC) in Berlin

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