The purpose of this Pilot program is to provide seed money for research that has not yet reached the stage of maturity necessary for funding by traditional NIH mechanisms such as RO1s and PO1s. Such research ideas should be considered innovative and may potentially be high risk. These applications are funded for one or two years and have a maximum funding limit of $65,000 per year. Research areas deemed relevant to the scope of this program include, but are not limited to: 

  • Development of new gene therapy vectors for CF.
  • Study of inflammatory responses to vector delivery of CF affected organs.
  • Assessment of host cell/vector interactions and the mechanisms of transduction to CF affected cell types.
  • Cystic fibrosis related diabetes and pancreatitis.
  • CF hepatic, gallbladder, and intestinal disease.
  • Identification of stem cells targets for gene therapy and cell therapy applications in CF.
  • Development of surrogate endpoints for assessing gene transfer and complementation in vivo.
  • Pathophysiology of CF in any affected organ, except the lung.
  • Development of animal models to test CF gene therapies in vivo.