Marcelo Simon Sola

rAAV vectors platform for R&D purposes

The rAAV platform has been created at Imagine during the first semester 2018 by Marcelo Simon Sola.

There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivered vectors.

The number of clinical trials using AAV recombinant vectors (rAAV) derived from the Adeno-Associated virus (AAV) for in vivo gene transfers has increased considerably these past few years and shows the high efficiency of these vectors for gene transfer and gene therapy. The most attractive features of rAAV are the non-pathogenicity, the lack of viral coding sequences, and the fact that rAAV are self-replication defective, predominantly non-integrative and have the ability to infect a wild variety of cell types and animal species.

 

Missions and services provided :

Most in vitro and 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 rAAV platform is the production of research grade recombinant AAV vectors (rAAVs) and rAAV vectors associated with extracellular vesicles (Exo-rAAV) for research projects, and pre-clinical in vitro and in vivo studies (mice and large animal models as non-human primates) for the Institut Imagine teams as well as for other teams within the DIM.

This function will ensure the availability of rAAV vectors for the different projects that involve experiments with rAAV vectors for expression of therapeutic transgenes, reporter genes (GFP, luciferase), empty capsid particles (AAV particles with no genome inside), or other constructs.  

The platform also aims the development of screening assays for the detection of pre-existing antibodies directed against the rAAV vector capsid of choice, in animal models and human subjects.

 

Equipment : Roller bottle incubator with 100  bottles capacity/run