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Thérapie génique non-virale de la mucoviscidose : évaluation des voies d'administration et adaptation des formulations

Abstract : Cystic fibrosis is a genetic disease with lung damages as the current main causes of death. Due to the absence or dysfunction of the CFTR chloride channel, CF patients have hyper-viscous mucus, particularly in the respiratory tract. This mucus is an environment favorable to infection development by opportunistic bacteria such as Staphylococcus aureus or Pseudomonas aeruginosa. The chronicity of infections coupled with significant inflammation leads to the progressive degradation of respiratory functions. Currently, apart from the heart-lung transplantation, no cure is still available for all patients.The approach by gene therapy appears to be a good strategy to cure all patients regardless of the type of mutations they have. It is a matter of bringing a healthy copy of the CFTR gene into the cells so that they express a functional protein. To do this, many barriers must be overcome. Among them, the presence of bacteria in the cellular environment is a brake against the transfer of genes in particular by vectors. It seems pertinent to develop a multifunctional formulation that on the one hand eliminates surface bacteria and on the other hand transfect the target cells. This formulation must remain effective after it has been aerosolized. During this work, several formulations, incorporating cationic lipids and silver compounds, have been developed.
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Submitted on : Tuesday, April 10, 2018 - 10:49:29 AM
Last modification on : Wednesday, October 14, 2020 - 3:52:16 AM


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  • HAL Id : tel-01762492, version 1



Angélique Mottais. Thérapie génique non-virale de la mucoviscidose : évaluation des voies d'administration et adaptation des formulations. Médecine humaine et pathologie. Université de Bretagne occidentale - Brest, 2017. Français. ⟨NNT : 2017BRES0146⟩. ⟨tel-01762492⟩



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