Non-viral gene delivery systems for gene therapy and vaccination

Séminaires & Conférences Chimie École Doctorale 459
Jeudi 21 Juillet 2016, 14h
Salle de Cours SC-16.01 – UM Faculté des Sciences (campus Triolet)
Non-viral gene delivery systems for gene therapy
and vaccination: design and biological activities
of histidylated chemical vectors
Prof. Chantal PICHON
Centre de Biophysique Moléculaire, UPR 4301 CNRS, 45071 Orléans cedex 02 ;
Université d’Orléans
courriel : [email protected]
(cette conférence sera présentée en français)
Recent clinical trials of gene therapy unambiguously demonstrate the potentiality
of this innovative therapy to cure diseases related to genetic disorders. Even though
viral delivery systems remain the best vehicles to introduce nucleic acids into cells;
there are some adverse events that raise serious safety concerns. Therefore, clinical
developments still require the use of alternative approaches of high safety, low
immunogenicity and easy manufacture. Efforts have been carried out to design
chemical gene delivery systems that incorporate viral-like features required for
efficient cell transfection. Nucleic acids therapeutics are nanosized particles made of
self-assembled nanometric complexes resulting from interactions between nucleic
acids and chemical vectors. These nanosized particles are quite different to
conventional drug delivery formulation.
I will present during this talk, the development of nucleic acids basednanoparticles made with our original histidylated polymers and lipids. I will show
mainly our data concerning mRNA based-vaccination to illustrate their efficiency. We
succeeded to design specific formulations that are targeted to spleen upon systemic
administration. They were active as prophylactic and therapeutic anti-cancer vaccines.
Nowadays, mRNA vaccines constitute a potential alternative to proteins and
inactivated microorganisms as shown by recent clinical trials data.