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Chitosan-Based Polymeric Nanoparticles as an Efficient Gene Delivery System to Cross Blood Brain Barrier: In Vitro
Ishaq N Khan1,2, Shiza Navaid3, Walifa Waqar4
1MIT Media Lab, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Abstract:
Significant progress has been made in the field of gene therapy, but effective treatments for brain tumors remain challenging due to their complex nature. Current treatment options have limitations, especially due to their inability to cross the blood-brain barrier (BBB) and precisely target cancer cells. Therefore options that are safer, more effective, and capable of specifically targeting cancer cells are urgently required as alternatives. This current study aimed to develop highly biocompatible natural biopolymeric chitosan nanoparticles (CNPs) as potential gene delivery vehicles that can cross the BBB and serve as gene or drug delivery vehicles for brain disease therapeutics. The efficiency of the CNPs was evaluated via in vitro transfection of Green Fluorescent Protein (GFP)-tagged plasmid in HEK293-293 and brain cancer MG-U87 cell lines, as well as within in vivo mouse models. The CNPs were prepared via a complex coacervation method, resulting in nanoparticles of approximately 260 nm in size. In vitro cytotoxicity analysis revealed that the CNPs had better cell viability (85%) in U87 cells compared to the chemical transfection reagent (CTR) (72%). Moreover, the transfection efficiency of the CNPs was also higher, as indicated by fluorescent emission microscopy (20.56% vs. 17.79%) and fluorescent-activated cell sorting (53% vs. 27%). In vivo assays using Balb/c mice revealed that the CNPs could efficiently cross the BBB, suggesting their potential as efficient gene delivery vehicles for targeted therapies against brain cancers as well as other brain diseases for which the efficient targeting of a therapeutic load to the brain cells has proven to be a real challenge.
Insights
Chitosan nanoparticles (CNPs) show promise for brain disease gene therapy by effectively crossing the blood-brain barrier (BBB). These biocompatible nanoparticles offer improved safety and higher transfection efficiency for targeted brain cancer treatments.
Area of Science:
- Biotechnology
- Nanomedicine
- Gene Therapy
Background:
- Gene therapy for brain tumors faces challenges due to the blood-brain barrier (BBB).
- Current treatments lack precise targeting and efficient delivery across the BBB.
- Safer, more effective, and targeted delivery systems are needed for brain disease therapeutics.
Purpose of the Study:
- To develop biocompatible chitosan nanoparticles (CNPs) as gene delivery vehicles.
- To evaluate the ability of CNPs to cross the BBB for brain disease treatment.
- To assess the safety and transfection efficiency of CNPs in vitro and in vivo.
Main Methods:
- Chitosan nanoparticles (CNPs) were prepared using a complex coacervation method.
- In vitro transfection efficiency was tested using Green Fluorescent Protein (GFP) in HEK293 and MG-U87 cell lines.
- In vivo studies were conducted in Balb/c mice to assess BBB crossing and efficacy.
Main Results:
- CNPs demonstrated superior cell viability (85%) compared to chemical transfection reagents (72%).
- CNPs exhibited higher transfection efficiency in vitro (53% vs. 27% via FACS).
- In vivo studies confirmed that CNPs can efficiently cross the blood-brain barrier.
Conclusions:
- Highly biocompatible CNPs are effective gene delivery vehicles for brain diseases.
- CNPs offer a promising alternative for targeted brain cancer therapy.
- The ability of CNPs to cross the BBB opens new avenues for neurological treatment.
