Sq-siRNA nanoparticles for Charcot-Marie-Tooth disease: formulation and colloidal stability in serum albumin solutions
In the framework of the Nanoprotection Labex program, the squalenoyled concept for the fragile macromolecules (siRNA) is used to preserve their efficacies and propose a new treatment of the Charcot-Marie-Tooth disease type 1A (CMT-1A). In this proposal, we aim to fully characterize the nanoparticles suspensions of Sq-siRNA after their formation and their evolution when incubated in different biological media. This is a mandatory step to control the formulation and to obtain first insight on the mechanism of the siRNA's pharmocinetics after injection. In addition, this new investigation will determine if our first results on Squalene adenosine nanoparticles suspension incubated in BSA solutions could be extrapolated to a macromolecular conjugate of a squalenoyl compound.
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TESTARD; Frédéric Gobeaux; GRILLO Isabelle; GUENOUN and Jean-Philippe Renault. (2018). Sq-siRNA nanoparticles for Charcot-Marie-Tooth disease: formulation and colloidal stability in serum albumin solutions. Institut Laue-Langevin (ILL) doi:10.5291/ILL-DATA.9-13-781
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