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. Preliminary results indicated that some synthesized nanoparticles indeed exhibited promising uptake in differentiated C2C12 myotubes indicating their potential effectiveness in muscle-targeting. Importantly, the nanoparticles demonstrated stability in their size and other key characteristics. However, further experimental work is required to achieve better selectivity and confirm their therapeutic potential. By synthesizing and evaluating these innovative nanoparticles, this thesis contributes to advancing gene delivery systems, offering hope for more effective treatments for DMD and similar muscular disorders. |
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Relatori: | Valentina Alice Cauda |
Anno accademico: | 2024/25 |
Tipo di pubblicazione: | Elettronica |
Numero di pagine: | 125 |
Soggetti: | |
Corso di laurea: | Corso di laurea magistrale in Ingegneria Biomedica |
Classe di laurea: | Nuovo ordinamento > Laurea magistrale > LM-21 - INGEGNERIA BIOMEDICA |
Aziende collaboratrici: | Institut Químic de Sarrià - CETS Fundació Privada |
URI: | http://webthesis.biblio.polito.it/id/eprint/33756 |
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