AAVs are efficient therapeutic platforms for the treatment of genetic diseases. Yet, past and ongoing clinical trials have highlighted some limitations, including the need for high doses to achieve therapeutic benefit, and the off-target transduction of various tissues.
With the growing success of gene therapy clinical trials, adeno-associated virus vector (AAV) manufacturing and quality control requirements tend to increase. Our group aims at improving current large scale viral vector production systems, such as the Sf9/Baculovirus expression system. We also develop novel analytical tools, based on next-generation sequencing (NGS), allowing us to identify and quantify the acid nucleic content of AAV particles.
Our group also focuses on the development of novel, chemically modified AAV particles, with increased therapeutic effect, at the interface between vectorology and chemistry. Chemically modified AAVs should enable to greatly decrease off-target effects, hence maximizing transduction in tissues of interest and improving the therapeutic index in AAV-based gene therapy.

Team members :

David Deniaud, Associate professor (Corail Team - CEISAM laboratory UMR CNRS 6230)
Mathieu Mével, PhD, Senior scientist (Associate researcher, Corail Team - CEISAM laboratory UMR CNRS 6230)
Magalie Penaud-Budloo, Post-doc
Dimitri Alvarez-Dorta, Post-doc (20% UMR INSERM 1089 - 80% UMR CNRS 6230)
Emilie Lecomte, Engineer
Mohammed Bouzelha, Engineer
Aurelien Leray, PhD student (50% UMR INSERM 1089 - 50% UMR CNRS 6230)
Pierre-Alban Lalys, PhD student (20% UMR INSERM 1089 - 80% UMR CNRS 6230)
Marianne Laugel, PhD student