Gene Therapy: The Role of Cytoskeleton in Gene Transfer Studies Based on Biology and Mathematics
Maria G. Notarangelo, Roberto Natalini and Emanuela Signori
Affiliation: Laboratory of Molecular Pathology and Experimental Oncology, CNR-IFT, via Fosso del Cavaliere 100, 00133, Rome, Italy.
Gene therapy is a promising approach for treating a wide range of human pathologies such as genetic disorders
as well as diseases acquired over time. Viral and non-viral vectors are used to convey sequences of genes that can be expressed
for therapeutic purposes. Plasmid DNA is receiving considerable attention for intramuscular gene transfer due to
its safety, simplicity and low cost of production. Nevertheless, strategies to improve DNA uptake into the nucleus of cells
for its expression are required. Cytoskeleton plays an important role in the intracellular trafficking. The mechanism regulating
this process must be elucidated. Here, we propose a new methodological approach based on the coupling of biology
assays and predictive mathematical models, in order to clarify the mechanism of the DNA uptake and its expression into
the cells. Once these processes are better clarified, we will be able to propose more efficient therapeutic gene transfer protocols
for the treatment of human patients.
Keywords: Gene therapy, intracellular transport, mathematical models, microtubules, plasmid DNA.
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