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Stem Cells and Neurodegenerative Disorders

Research Coordinator

Dr. Charo Sánchez Pernaute

The goal of this translational research program is to further understanding and improve treatment of neurodegenerative diseases using stem cell technology. Within this framework, human pluripotent stem cells are employed in various projects to obtain neurons and other relevant cell types involved in degenerative processes of the central and peripheral nervous system like Parkinson Disease and Neuropathic Pain. Human pluripotent stem cells provide unprecedented means to study degenerative disorders of the nervous system from a completely new angle, as it is now possible to generate and manipulate specific types of human neurons in vitro. Using reprogramming techniques to generate iPS cell lines and induced neurons, relevant cell types can be obtained from patients and at risk individuals with neurodegenerative disorders.

In Inbiomed we are working to apply these promising technologies to advance the knowledge of neurodegenerative disease mechanisms, develop disease-modifying treatments and restore the function of affected neural circuitry. Investigators in the department have expertise in human pluripotent stem cells, basic neuroscience, electrophysiology, in vivo models and clinical neuropathology.

Current projects aim to

1. investigate the patterning and specification of human central and peripheral neuronal subtypes in order to identify factors and mechanisms conferring disease vulnerability to various neuronal populations
2. create in vitro disease models to characterize pathogenic mechanisms and pathways, discover novel therapeutic targets and evaluate therapeutic candidates
3. establish adequate cell sources for cell replacement and restorative therapies

Collaborations

Biodonostia and Hospital Donostia, Neurogenetics Unit: Dr A López de Munain.

University of the Basque Country (UPV), Medical School: Prof Luisa Ugedo.

Neurogenesis and Neural Cells Consortium (funded by kutxa Obra Social).

• CIPF, Valencia: Prof José Manuel García Verdugo.
• CIMA, University of Navarra: Dr María Rosario Luquin.

University of Harvard, HMS, McLean Hospital, Belmont, MA: Dr Kai Sonntag.

Sloane Kettering Institute, NYC, NY: Dr Lorenz Studer.

CIC-BioGune, Bilbao: Dr Jokin Castilla.