Stem Cell Delivery of TTP1 in Batten Disease Dogs

Rebecca Whiting, PhD

Rebecca Whiting, PhD, of the University of Missouri discusses her recent study showing improvement in visual function in dogs given TTP1 via stem cells.


Whiting REH, Tracy CJ, Pearce JW, et al.  Retinal function and structure are preserved in a canine model of CLN2 Batten disease after intravitreal implantation of stem cells genetically modified to overproduce TPP1 enzyme. Presented at the 15th International Conference on Neuronal Ceroid Lipfuscinosis (Batten Disease); Boston, MA; October 5-8, 2016. Abstract O35.

This article was made possible by a sponsorship from BioMarin Pharmaceutical Inc. Rare Disease Report has sole control over the editorial content.
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