Xavier Bracco
Synthesis, characterization and in vitro selectivity study of Zwitterionic and Peptide-Enhanced pBAES Nanoparticles for Targeted Gene Delivery in muscular cells.
Rel. Valentina Alice Cauda. Politecnico di Torino, Corso di laurea magistrale in Ingegneria Biomedica, 2024
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Abstract
Duchenne muscular dystrophy is a severe genetic disorder characterized by progressive degeneration of muscles due to mutations in the dystrophin gene. Even if corticosteroids and gene therapy have been pursued as treatments, there is still no clear cure. One of the major challenges with DMD gene therapy involves efficient delivery of therapeutic genes into muscle cells while avoiding rapid immune mediated clearance and ensuring long-term gene expression. This thesis describes the development of nanoparticle-based gene delivery systems using zwitterionic polymers as a promising non-viral strategy for targeted muscle delivery. Such nanoparticles are developed to extend their circulation time and thereby enhance their bioavailability and improve targeting to muscle cells, addressing key challenges in gene therapy for DMD.
Incorporating muscle-targeting peptides into the nanoparticle design further enhances delivery precision, improving transfection efficiency and minimizing off-target effects
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