Jose L. Lanciego
Center for Applied Medical Research (CIMA and CIBERNED), Spain
Title: NEW GENE THERAPY APPROACHES FOR PARKINSON’S DISEASE
Biography
Biography: Jose L. Lanciego
Abstract
The field of Gene Therapy in the CNS has recently witnessed a number of major conceptual changes. Besides the traditional thinking that comprises the use of viral vectors for the delivery of a given therapeutic gene, a number of original approaches have been recently envisaged, focused on using vectors carrying genes to further modify brain circuits of interest. It is expected that these approaches will ultimately induce a therapeutic potential being sustained by induced changes in brain circuits. Here we will illustrate the rationale behind several experiments that are currently under implementation in the non-human primate (NHP) model of Parkinson’s disease (PD). Among others, we will focus on the following approaches: (i) in vivo reconstruction of the nigrostriatal pathway, (ii) selective elimination of hyperactive basal ganglia circuits in dyskinetic macaques, and (iii) strategies for in vivo reprogramming of striatal neurons. Besides considering the translational potential of these approaches, we hope that these experiments, complementary to each other, will allow us to generate new data supporting a better understanding of the pathophysiology of PD. Supported by grants from the European Research Council (ERC Advanced grant) and by CoEN-Pathfinder.