About the book
Any mutation in the genome can cause a vast number of diseases, and the treatment of these diseases at the gene level is a very recently emerging approach in clinical practice with promising future implications. Gene therapy is described as the transfection of the therapeutic gene (DNA or RNA) either to the cytoplasm (RNA) or the nucleus (DNA) for curing various acquired and inherited diseases. This transfection can selectively improve or inhibit (antisense) gene function. As degradation of nucleic acids can occur during cellular uptake, which prevents the effective penetration of naked DNA or RNA into the cell, an appropriate carrier is needed.
There are two main sorts of carriers which are called vectors: viral or non-viral vectors. Although viral vectors exhibit higher transfection efficiencies compared to non-viral based delivery, their adverse effects such as immunogenicity, oncogenicity, and cytotoxicity may cause limitations in clinical use. To overcome these drawbacks, non-viral vectors including, but not limited to polymers, lipids, and inorganic nanoparticles have recently drawn vast attention in gene therapy. This book aims to provide the reader with a comprehensive overview of the current state-of-the-art in non-viral vectors focusing on the potential employment of several nanomaterials which can be used in gene therapy.