Positron emission tomography reporter gene strategy for use in the central nervous system

With any new treatment it is imperative the medical community has a way to monitor its effectiveness, tracking its location, monitoring its expression, and observing off-target effects. Gene/cell therapy is no exception. The growing number of studies creates a need to monitor them more accurately. A number of PET reporter gene systems have been reported; however, they have limitations, including regions of high endogenous gene expression in the central nervous system (CNS), low specificity, or endogenous expression of the reporter gene in microglia. One of these systems has been used in the clinical setting; however, its associated imaging agent lacks blood-brain barrier penetration. Our approach aims to overcome these limitations and facilitate monitoring of gene/cell therapy in the CNS.