Reconstituting functional neurofibromin by delivering the correct version of the gene to cells in the NS presents a promising treatment that has high potential for clinical translation. However, this has been challenging due to (1) the difficulty of encapsulating and delivering the large NF1 gene, which is out of reach of viral vectors since they have a small cargo capacity, and (2) the lack of safe targeting strategies that can preferentially deliver therapeutics to cells in the nervous system. To overcome these challenges, our team from Battelle and University of Alabama, Birmingham, proposes to use synthetic polymer nanoparticles (PNP) to encapsulate and deliver the NF1 gene to cells in the nervous system. The approach, to simultaneously test a large variety of delivery candidates at an early stage, both in vitro and in vivo, will help us avoid the pitfalls of conventional drug development cycles and identify delivery vector candidates with high promise for clinical success.
Investigators
Cherry Gupta, PhD
Battelle Memorial Institute
Cherry Gupta, PhD
Battelle Memorial Institute