Pr. Marina Cavazzana & Federico Mingozzi
Most in vivo experiments aimed at developing AAV-based gene therapies for neuromuscular, neurological and metabolic diseases require production of vectors of high quality and sufficient quantity. The goal of this project is the development of a vector Core Facility for the production of research grade AAV vectors for research projects, and pre-clinical in vitro and in vivo studies (mice and large animal models such as non-human primates) for the Institut Imagine teams as well as for other teams of the Paris Region. This function will ensure the availability of recombinant AAV vectors for the different projects that involve experiments with AAV vectors for expression of therapeutic transgenes, reporter genes (GFP, luciferase), empty capsid particles (AAV particles with no genome inside), or other constructs. This platform will be located within the Institut Imagine.