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- Title
Customizing Cationic Lipid Nanoparticles for Promising Gene Therapy Applications.
- Authors
Jummah, Nurul; Satrialdi; Artarini, Aluicia Anita; Mudhakir, Diky
- Abstract
Introduction: Gene therapy is a medical approach aimed at treating or preventing diseases by addressing fundamental genetic irregularities. Unlike conventional approaches reliant on drugs or surgical interventions, gene therapy employs innovative techniques to rectify genetic anomalies, including gene mutations. This therapeutic approach holds great promise, particularly in its capacity to precisely target the nucleus through the utilization of cationic lipid nanoparticle (cLNP) delivery systems. Methods: In this study, we formulated cLNPs using an emulsification methodology. The attributes of cLNPs, including size, polydispersity index, zeta potential, and morphological structure, were assessed using transmission electron microscopy. Entrapment efficiency was also being performed. For cLNP-mediated transfection, incorporation of cationic lipid 1,2-dioleoyl-3-trimethylammonium-propane (DOTAP) enabled electrostatic interactions with nucleic acids. To gauge the penetration ability of cLNPs into TM4 cells and their subsequent nucleus targeting, enhanced green fluorescent protein (EGFP) was used as a marker protein. EGFP served as confirmation for cLNP's efficacy in delivering the pEGFP gene into the nucleus, where the expression of EGFP protein is visually observed via confocal microscopy. Results: With a particle size of 146.17±33.47 nm and a positive particle charge of +0.43 mV, the cLNPs showed remarkable ability to penetrate TM4 cells. The polydispersity index showed exceptional size uniformity, with a value of 0.313±0.022 and entrapment efficiency of 90.03±0.055%. The TEM results showed the spherical morphology of these cLNPs, confirming their robust structural integrity. The interaction between materials forming the cLNPs was further evident through the difference in Tm, as observed in the DSC results. The cLNP were also able to internalise the nucleus, as indicated by the expression of EGFP resulting from the successful delivery of pEGFP by the cLNP into the cells. Conclusion: The characterization data of these cLNPs shows their potential as an efficacious gene therapy delivery system for addressing complex diseases within cellular contexts.
- Subjects
GENE therapy; GREEN fluorescent protein; CATIONIC lipids; NUCLEIC acids; GENETIC techniques; TRANSMISSION electron microscopy
- Publication
Malaysian Journal of Medicine & Health Sciences, 2024, Vol 20, p42
- ISSN
1675-8544
- Publication type
Abstract