Functionalized liposomal nanoparticles for efficient gene delivery system to neuronal cell transfection

Area of Science:

• Biotechnology
• Nanomedicine
• Neuroscience

Background:

• Liposome-based delivery systems offer potential for treating neurodegenerative diseases.
• Targeted delivery to the brain is crucial for effective therapies.

Purpose of the Study:

• To characterize surface-modified liposomes (PenTf-liposomes) for targeted gene delivery to brain cells.
• To evaluate the efficiency, safety, and cellular uptake mechanisms of PenTf-liposomes.

Main Methods:

• Liposomes were surface-modified with transferrin (Tf) and penetratin (Pen).
• Encapsulation of plasmid DNA, in vitro release kinetics, cytotoxicity, and hemolytic assays were performed.
• Cellular internalization pathways (clathrin-mediated endocytosis) and gene transfection efficiency were assessed in various cell lines.

Main Results:

• PenTf-liposomes efficiently encapsulated DNA, protected it from degradation, and showed sustained release.
• The formulation exhibited low cytotoxicity and was non-hemolytic.
• Significantly higher reporter gene transfection and protein expression were observed with PenTf-liposomes compared to unmodified liposomes, likely due to efficient endosomal escape.

Conclusions:

• Rationally designed bifunctional PenTf-liposomes are an efficient tool for targeted gene delivery to neuronal cells.
• These liposomes utilize energy-dependent pathways, including clathrin-mediated endocytosis.
• PenTf-liposomes represent a safe and non-invasive approach for neuronal gene transfection.