If an MDI formulation can deliver DNA that remains viable after aerosolization, without any toxicity to cells from the formulation, MDIs could potentially become the delivery method of choice for gene therapy. This article describes a proof of concept method for doing so.
For some time, researchers have been investigating the possibility that certain lung diseases, including inheritable conditions like cystic fibrosis (CF) and malignancies such as carcinomas, could potentially respond to gene therapy delivered directly to the lung. Studies have already shown some success in correcting genetic defects. Pulmonary delivery of DNA could also provide an effective immunization against diseases such as influenza and measles, as that routemimics the natural route of infection.