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Séminaire Chimie ED459

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 | Université d’Orléans)

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Le Jeudi 21 Juillet 2016 à 14h
UM FdS, Salle de Cours SC-16.01

(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 based-nanoparticles 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.

Contact local ICGM : Dr. Jean-Olivier Durand, D.R. CNRS (équipe IMNO)

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