"Transgene Expression in the Murine Airways": Strategies to extend the duration of transgene expression using insulator elements, anti-inflammatory drug and zero-CpG plasmid DNA

Gene therapy can be described as the intracellular delivery of genetic material to generate a therapeutic effect by correcting an existing abnormality or providing cell with new function. As gene therapy attempts to treat the roots of the disease rather than the symptoms, it is heralded as one of the revolutionary treatments able to cure a myriad of genetic diseases. Cystic Fibrosis (CF) is an attractive target for gene therapy as it is a monogenic disease and the target organ is accessible for direct gene delivery. However, previous efforts to use gene therapy for CF have been disappointing due to the transient transgene expression in the lung. In this book, the author described and tested three strategies to improve the duration of transgene expression in the murine airways, in a non-viral gene delivery context. The three approaches analyzed were: (i) the incorporation of insulator elements in the expression cassette, (ii) the administration of immunosuppressive drug, and (iii) the use of zero-CpG plasmid DNA. This book should serve as a guide to research students, physicians and scientists interested in the improvement of the non-viral gene transfer method.