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Use of IPS cell-derived neurons to study a possible role for autophagy in the neurodegenerative phenotype of Cockayne Syndrome

Grant number: 12/04832-8
Support type:Scholarships abroad - Research Internship - Doctorate (Direct)
Effective date (Start): August 01, 2012
Effective date (End): July 31, 2013
Field of knowledge:Biological Sciences - Biochemistry - Molecular Biology
Principal Investigator:Carlos Frederico Martins Menck
Grantee:Alexandre Teixeira Vessoni
Supervisor abroad: Albert R. La Spada
Home Institution: Instituto de Ciências Biomédicas (ICB). Universidade de São Paulo (USP). São Paulo , SP, Brazil
Local de pesquisa : Sanford Consortium for Regenerative Medicine, United States  
Associated to the scholarship:11/13433-7 - Autophagy analysis in response to agents capable of inducing DNA damage, BP.DD

Abstract

This project aims at studying a possible role for autophagy in the neurodegenerative phenotype observed in patients with Cockayne Syndrome. In order to do that, human skin fibroblasts from CS patients will be reprogrammed to a pluripotent state (iPS cells) and then, differentiated into neurons, thus, constituting a model to study the neurodegenerative phenotype observed in these patients. Steady-state levels of autophagy/mitophagy, as well as in response to oxidative stress (which is believed to play an important role in this phenotype), will be analyzed in these cells. The study of autophagy in this model is based on observations that this process is strictly related to neurodegenerative disorders such as Alzheimer's, Parkinson and Huntingtion diseases. (AU)