Aurélien LERAY, PhD student in CEISAM CORAIL team, will defend her thesis entitled “Chemically modified AAV for gene therapy” on February 16th 2021 at 14h in CEISAM’s conference room “Marie Curie”.
AAV, or adeno associated virus, are now becoming therapeutic products thanks to their properties. The hundreds of ongoing clinical trials demonstrated their efficiency and gene therapy treatments as Glybera, Luxturna and Zolgensma are now commercialised. However, clinical trials revealed the limitation of the use of AAV as a gene therapy vectors. AAV have a broad distribution, possess a low efficacy at low doses due to neutralizing antibodies. To overcome those barriers genetic engineering is widely used. Our team developed an alternative, the chemical modification of lysine residue at the surface of AAV. Results showed a redirection of the vector tropism, a loss of antibodies recognition and an overall increase of efficiency. In this thesis, we seek to chemically modify tyrosine residues at the surface of AAV vectors to enhance its efficacy. Synthesis and coupling on the vector of synthetized compounds and the properties of the chemically modified vectors will be studied.
COVID Information :
For reasons of hygiene, security and confidentiality, the defense will take place behind closed doors.