Biodegradable Polymeric Nanoparticles for Gene Delivery

Increasingly, scientists and clinicians are discovering the genetic basis of diseases, ranging from monogenic disorders to multigenic diseases such as various cancers. Moreover, human diseases are increasingly being understood on the molecular level, rather than as a phenotype, which is moving treatments towards stratified and personalized medicine. Gene therapy holds the promise of a technology that could address this growing need for genetic medicine as it can potentially tune individual cell gene expression on or off in a targeted and precise manner. The technology could, theoretically, also be applied to almost any human disease. The central challenge is that in practice, safe and effective delivery of desired nucleic acids to targeted human cells is very difficult. This chapter outlines these challenges in gene delivery and discusses state-of-the art approaches using biodegradable polymers to overcome these obstacles and obtain successful gene delivery in vitro and in vivo. Biodegradable gene delivery polymers, or plastics that are designed to safely deliver a biological cargo inside cells and then degrade, have certain advantages over other materials and viruses for the delivery of genes. This chapter elucidates the diverse types of biodegradable polymers used for gene delivery, the related nanoparticulate systems they form with nucleic acids, and the structural properties that increase their efficacy and safety.