Objective 1. To generate in vitro disease model by creating ARSACS disease- specific cerebellar cells via iPSC from patients and healthy individuals as controls which will serve as a tool to depict disease-specific molecular markers.
Objective 2. The comparative functional analysis between control and patient derived cerebellar cells (Purkinje and granule cells) will include molecular, cellular studies focused on detection of cerebellar specific markers through the differentiation and cerebellar electrophysiological recordings
Duration: one year
Grant: $100,000
Contact:
Dr.Slaven Erceg
Dr.Slaven Erceg, Head of Stem Cell Therapies in Neurodegenerative Diseases
Research C enter “Principe Felipe”, Valencia (Spain)
Email: serceg@cipf.es