11.2022 - 02.2026

Preclinical models for the physiopathological transition

Murine muscle cells (C2C12) were transfected with human expansions (CTGs) to establish an in vitro model of myotonic dystrophy type 1. The cells were treated with various types of nanocarriers (pegylated and non-pegylated liposomes, pegylated and non-pegylated PLGA nanoparticles, nanohydrogels) loaded with pentamidine, a drug capable of reducing nuclear alterations induced by genetic mutations in DM1 but at concentrations incompatible with life. No pentamidine-laden nanocarriers have been shown to reduce cell viability even over long timescales, but transmission electron microscope analyzes showed damage to cells treated with non-pegylated liposomes and non-pegylated PLGA nanoparticles, which were therefore excluded from the trial. The remaining nanocarriers were tested for their effectiveness in reducing nuclear foci (DM1 marker) with promising results. A manuscript is being written. 

The results of this basic research can pave the way for the application of nanocarriers for the treatment of muscle genetic pathologies.

Settori Scientifico Disciplinari

BIO/16  HUMAN ANATOMY

Spoke 4 : Perception and Brain-Body Interaction