Development of biomaterials for gene therapy

Area of Science:

• Biomaterials Science
• Gene Therapy
• Nanotechnology

Background:

• Viral vectors face challenges in safety, immunogenicity, and mutagenesis for gene therapy.
• Polymeric gene carriers present a promising alternative to viral vectors.
• Effective polymer-based gene therapy requires control over cellular uptake and intracellular trafficking.

Purpose of the Study:

• To review the conceptual and experimental aspects of polymer-based gene delivery.
• To provide an overview of recent advancements in polymers for enhancing plasmid-based gene therapy.
• To highlight the potential of polymeric carriers as commercially viable gene medicines.

Main Methods:

• Review of recent literature on polymer-based gene delivery systems.
• Analysis of conceptual and experimental data on polymeric gene carriers.
• Exploration of strategies to improve cellular access, uptake, and nuclear delivery of plasmid DNA.

Main Results:

• Polymeric gene carriers can overcome safety and immunogenicity issues associated with viral vectors.
• Current limitations in polymer-based gene therapy include inefficient endosomal release, cytoplasmic transport, and nuclear entry.
• Recent designs aim to enhance the effectiveness of polymer-mediated plasmid delivery.

Conclusions:

• Polymeric gene carriers show significant potential for treating genetic and acquired diseases.
• Further research is needed to address current limitations for optimal therapeutic outcomes.
• Polymeric carriers represent a promising avenue for the development of commercial gene medicines.