IPs differentiation into neurons

F.J. PITOSSI

Congreso; Asociación Brasilera de Terapia celular; 2015

The appearance of human iPS cells has changed the perspectives of human cell therapy. This new technology paves the way not only for new cell therapies but also for disease modeling and drug screening. One main bottleneck for the application of this technology is the generation of cells with specific functions from iPS cells. We will discuss the challenges associated with the generation of neurons from iPS cells with data from two lines of research: 1. In vitro modeling of epilepsy and 2. Cell therapy for Parkinson ´s Disease. In the first case, glutamatergic and gabaergic neurons were generated from iPS obtained from fibroblasts from patients with Focal Benign Childhood Epilepsy and related and unrelated controls. Neurons from epileptic patients acquired distinct features such as shorter axons and delayed electrophysiological properties compared to controls, corroborating the usefulness of these cells for in vitro modeling of disease. In the second project, human dopaminergic neurons were generated in collaboration with the group of X. Zeng at the Buck Institute in California. These and control cells were transplanted in a pre-clinical model of Parkinson ´s Disease (PD), under immunosuppression. The animals receiving dopaminergic neurons showed motor recovery together with persistent neuroinflammation. These results showed the therapeutic potential of these cells for PD treatment.