Development of in vivo gene therapy methods to treat Parkinson’s disease.f

Our main objective in this project is to develop cell-specific lentiviral vectors that can be regulated and used to treat the hallmarks features of Parkinson´s disease, and research tools for the further understanding of Parkinson´s disease.

We will investigate the differential expression miRNAs in the striatum and use this data to develop vector s specific for subpopulations of neurons. By over expression of RET we  will reinstate the GDNF response in  the alpha-synuclein model of Parkinson´s disesae. We will investigate the mechanisms of the rescue by the use of spatial transcriptomics.

Saturated autophagy has been implicated as a major mechanisms for cell death in Parkinson´s disease.

We will adress this  by CRISPR activation of  endogenous TFEB expression to increase the activity of autophagy in the dopmine cells of the substantia nigra.

The projects will result in scientific tools that will be useful to address fundamental questions about basal ganglia physiology as well as be used to develop new therapies for PD.