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Updated: Dec 1, 2025
Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry
Published on: March 1, 2013
Cellular gene delivery via poly(hexamethylene biguanide)/pDNA self-assembled nanoparticles
Alexandru Chivu1, Kantaraja Chindera2, Graça Mendes1
1Centre for Biomaterials in Surgical Reconstruction and Regeneration, Division of Surgery & Interventional Science, University College London, Rowland Hill Street, London NW3 2PF, United Kingdom.
Poly(hexamethylene biguanide) (PHMB) effectively delivers plasmid DNA and oligodeoxynucleotides into cells as nanoparticles. This novel gene delivery vector shows comparable or superior efficiency to PEI, with potential for clinical gene therapy applications.
Area of Science:
- Biotechnology
- Nanotechnology
- Molecular Biology
Background:
- Gene therapy holds promise for treating genetic diseases, but efficient intracellular delivery of genetic material remains a significant challenge.
- Polycationic vectors are widely explored for gene delivery, yet face barriers like cellular uptake and endosomal escape.
- Developing safe and effective non-viral vectors is crucial for advancing gene therapy towards clinical applications.
Purpose of the Study:
- To investigate poly(hexamethylene biguanide) (PHMB) as a novel nano-vector for intracellular delivery of plasmid DNA (pDNA) and oligodeoxynucleotides (ODNs).
- To characterize the self-assembled PHMB-nucleic acid nanoparticles and evaluate their gene delivery efficiency in model cell lines.
- To compare the performance and safety profile of PHMB-based vectors against established polycationic vectors like polyethyleneimine (PEI).
Main Methods:
- Self-assembly of PHMB with pDNA or ODNs into nanoparticles at pH 7.4 and 12.
- Characterization of nanoparticle size, charge, and morphology.
- Assessment of cellular uptake and gene expression levels in HepG2, HEK293T, and HeLa cell lines.
- Comparison of PHMB-based vector performance with PEI analogues.
Main Results:
- PHMB formed stable nanoparticles with pDNA and ODNs at different pH values.
- The nanoparticles demonstrated efficient cellular uptake across multiple model cell lines.
- Measurable gene expression levels were achieved, comparable to or exceeding those obtained with PEI.
- PHMB exhibited a favorable safety profile, consistent with its known properties.
Conclusions:
- PHMB serves as an effective nano-vector for intracellular delivery of pDNA and ODNs.
- PHMB-based nanoparticles offer a promising alternative to existing gene delivery systems.
- The safety profile and efficacy of PHMB warrant further investigation for potential clinical translation in gene therapy.
