| dc.description.abstract | ABSTRACT :
Neurodegenerative diseases are a group of progressive and degenerative disorders that affects the nervous
system and causes various symptoms in patients with these pathologies. Unfortunately, there is no cure for these
diseases, so treatment aims to alleviate symptoms, prevent complications, and slow the progression. In this
research, we propose a carrier nanosystem that uses spherical silica nanoparticles functionalized with the toxin
ligand domain derived from Clostridium botulinum toxin (BTXC), which binds with high affinity with the SV2
motoneuron´s receptor, so this nanosystem could reach the nervous system through retro-axonal transport
Silica nanoparticles were synthesized through the Stöber method, obtaining negative charge particles that will
bind electrostatically with the histidine tails (positive charge) of the toxin ligand domain. BTXC was overexpressed,
extracted, and purified from E. coli. Nanoparticles loaded with FITC were functionalized and added to motoneurons like cell cultures (NSC34) to determine if the toxin ligand domain gives the nanoparticles specificity for neurons. Flow
cytometry demonstrated that the BTXC ligand increased the affinity of nanoparticles for motoneurons-like cells. In
addition, to evaluate the gene delivery ability of this nanosystem, nanoparticles that contain the DNA were
synthesized, then functionalized with BTXC and added to NSC34 cells, the gene expression was evaluated by
fluorescence microscopy and flow cytometry.
These results indicate that BTXC functionalization gives these carrier nanosystems affinity for motoneurons-like
cell cultures. Furthermore, they have shown the ability to encapsulate biological material inside, which would be an
important gene therapy for future neurodegenerative disease treatment. | es_ES |
