FigureĀ 1.

FigureĀ 1. From: Pluripotent stem cells in neurodegenerative and neurodevelopmental diseases.

iPSC to model neurodegenerative and neurodevelopmental diseases. Human iPSC from neurologic patients and controls are generated after somatic tissue reprogramming (e.g. skin or blood cells). Neural progenitor cells (NPC) are generated and are further differentiated into neurons and/or glial cells. Neurons are then differentiated into subtypes of neurons such as dopaminergic, cholinergic, etc. Cellular phenotype is assessed by measuring neuronal morphology (i.e. process branching, spine density/size/maturation). Next, connectivity and circuitry integration can be analyzed by calcium influx transients, electrophysiology and transneuronal tracing with the rabies virus. In addition, the cross-talk between neurons and glia can be studied to tease out autonomous and non-autonomous aspects of the disease. Once a distinct disease-related phenotype is identified, drug-screening platforms can be developed to test compounds that improve cellular phenotype. Therapeutic compounds could emerge from the screenings, potentially benefiting neurologic patients.

Maria C.N. Marchetto, et al. Hum Mol Genet. 2010 April 15;19(R1):R71-R76.

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