Douglas Ballon

My group has developed several imaging and gene delivery techniques for application to an autosomal recessive pediatric neurodegenerative disease known as late infantile neuronal ceroid lipofuscinosis (CLN2 disease). Specifically, we developed the first non-invasive biomarker panel for CLN2 using multiparametric MRI. This work was featured in an invited presentation to the international WORLD Symposium on lysosomal storage diseases in 2011, and the methods were further developed for inclusion into a currently ongoing worldwide multi-Center trial of the first drug that has been shown to be successful against CLN2. In 2014, we presented the first demonstration of adeno-associated viral vector mediated intraarterial gene delivery and expression for LINCL in the mouse brain. This work was originally presented at the New York Academy of Sciences conference on the Blood Brain Barrier in October 2011 where it received a Best Poster Award and a special oral presentation at the conclusion of the conference. Most recently, we developed robust reproducible viral vector radiolabeling. A major problem in viral vector mediated gene therapy is the issue of tropism, or the ultimate destination of administered vector in the body. Iodine-124 labeling of adeno-associated virus capsids was achieved that allows for monitoring of the spatial distribution of vector in the CNS. Adeno-associated virus is one of the most useful viral vectors in gene therapy trials. I was the senior investigator on these studies. 

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