chapter  21
14 Pages

Hepatic delivery of nucleic acids using hydrodynamics-based procedure


INTRODUCTION Nucleic acids therapy using oligonucleotides (oligonucleotide therapy) or full coding sequence with regulatory elements for its expression (gene therapy) is a promising new modality in medical practice. This therapy intends to cure a disease through regulating the level of gene product(s), crucial for causing or maintaining a pathophysiological condition. In general, oligonucleotide therapy is designed to inhibit the synthesis of disease-related proteins, while gene therapy aims at correcting genetic defects by the import of a functional gene into target cells. As nucleic acid drugs are hydrophilic, large in molecular weight, and unable to cross the cell membrane freely, a delivery mechanism is often required for these genetic materials to reach their intracellular targets. Development of a safe and efficient method for nucleic acid delivery has been the focus of many studies in the past few years.