There are a number of areas of focus in the development of an effective and valuable treatment of Cystic Fibrosis. These include:
Plasmid DNA molecules are one of the most widely used tools in molecular biology research. They are derived from naturally occurring molecules in microbes. These circular double stranded DNA molecules have been manipulated by molecular biologists to allow for the propogation and study of interesting pieces of DNA from a wide variety of sources. Plasmid molecules are available to meet a wide range of needs.
The relative accessibility of the pulmonary epithelium makes aerosol delivery of gene therapy formulations an attractive possibility, allowing non-invasive application to target cells within the lung whilst minimising the risks associated with systemic delivery.
In parallel with the development of gene therapy products there has been the development of clinical assays to gauge the effectiveness of gene therapy. In many ways this type of research is as demanding as the development of the vectors themselves because without sufficiently powerful assays, it is impossible to determine the effectiveness of our technologies. The types of assays we have developed can be broken down into a number of groups.
Recombinant viral vectors are gene therapy vectors which are derived from viruses. Many viruses such as adenovirus, adeno-associated virus & Sendai virus have evolved over millions of years to become efficient agents at infecting human airway cells. Using an array of molecular biology tools, methods have been developed to allow virus particles to be modified such that they contain no infectious material or replicative capacity. Instead they package a gene therapy construct within their viral.
Lentiviral vector production is complex. Non viral expression plasmids are produced which encode the structural components of the virus and the packaging elements required to package the viral single stranded RNA into the final vector. The entire production process takes place inside human cells grown in culture.