Revolutionizing Gene Therapy: Biomaterials as Enabling Tools for Targeted Delivery and Enhanced Efficacy

Abstract

Bioengineered smart nano/biomaterials have emerged as promising tools for efficient and targeted gene delivery in gene therapy. Gene therapy has the potential to treat various genetic disorders, cancers, and infectious diseases by replacing, repairing, or regulating the expression of disease-causing genes. However, the success of gene therapy critically depends on the efficient delivery of therapeutic genes into target cells or tissues while minimizing off-target effects and toxicity. Bioengineered smart nano/biomaterials offer several advantages over conventional gene delivery vehicles, such as viral vectors and cationic lipids, including improved stability, biocompatibility, and tunable properties. This review provides an overview of bioengineered smart nano/biomaterials for gene delivery, including their design, fabrication, mechanisms of action, and applications in gene therapy. The design and fabrication of smart nano/biomaterials involve the synthesis and functionalization of various materials, such as polymers, lipids, peptides, and inorganic nanoparticles, to optimize their physicochemical properties and biological interactions. Mechanistically, smart nano/biomaterials can efficiently deliver DNA/RNA cargo into target cells by overcoming biological barriers, such as cellular membranes and endosomes, and facilitating nuclear entry and gene expression. Applications of smart nano/biomaterials in gene therapy include the treatment of genetic disorders, cancers, and infectious diseases, as well as targeted gene therapy for specific cell types or tissues. However, several challenges remain to be addressed before the clinical translation of smart nano/biomaterials for gene therapy, including safety, toxicity, and immunogenicity issues, optimization of delivery efficiency and specificity, and regulatory and ethical considerations. In conclusion, bioengineered smart nano/biomaterials hold great promise for the development of safe and effective gene therapies that can revolutionize the treatment of various diseases.