Developing non-viral or viral vectors for efficient and targeted delivery of genetic material, such as DNA or RNA, for gene therapy applications

Abstract

Gene therapy has emerged as a promising approach for treating a wide range of genetic and acquired diseases by introducing or modifying genetic material within cells. To achieve successful gene therapy, efficient and precise delivery of genetic material, such as DNA or RNA, to target cells is essential. This abstract explores the development of both non-viral and viral vectors for the delivery of genetic material in gene therapy applications. Non-viral vectors, including lipid nanoparticles (LNPs), polymer-based carriers, and cell-penetrating peptides, have gained significant attention due to their safety profile and ease of production. These vectors are designed to protect genetic material from degradation, facilitate cellular uptake, and release the cargo at the desired location. Recent advancements in nanotechnology have enabled the design of customizable non-viral vectors with enhanced delivery efficiency and reduced offtarget effects. Viral vectors, on the other hand, harness the natural infectivity of viruses to transport genetic material into target cells. Retroviruses, lentiviruses, adenoviruses, and adeno-associated viruses (AAVs) are commonly used viral vectors in gene therapy. Viral vectors offer high transduction efficiency but may trigger immune responses or pose risks of insertional mutagenesis. Efforts in vector engineering have led to the development of safer viral vectors with improved targeting capabilities and reduced immunogenicity. AAVs, in particular, have gained prominence due to their ability to achieve long-lasting gene expression with minimal adverse effects. Targeted delivery strategies aim to enhance vector specificity, ensuring that genetic material reaches the intended cell type or tissue. These strategies include modifying vector surface proteins, employing tissue-specific promoters, or utilizing ligand-receptor interactions. In conclusion, the successful application of gene therapy relies on the development of efficient and targeted delivery systems for genetic material. Non-viral and viral vectors offer distinct advantages and continue to evolve to meet the demands of gene therapy applications. Advances in vector design, safety, and targeting strategies hold promise for the continued progress of gene therapy as a transformative medical intervention.