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2018 BDSRA Whiting CLN2

Long-term delivery of TPP1 enzyme in the canine model of CLN2

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2018 BDSRA Whiting CLN2

  1. 1. CLN2 Long-term delivery of TPP1 enzyme in the canine model of CLN2 Rebecca Whiting, Martin Katz, Ophthalmology, School of Medicine, University of Missouri http://medicine2.missouri.edu/neurodegenerativediseases/ LONG TERM TREATMENT AFTER A SINGLE INJECTION  Preliminary results suggest that the same cells will be successful in treating the brain long term CONCLUSIONS  Stem cell delivery of TPP1 enzyme shows potential for providing TPP1 long-term after a single injection.  We are making progress towards the goal of a one-time curative treatment for CLN2 INTRODUCTION  Our lab continues working to optimize long-term delivery of TPP1 to the brain and eye using a dog model of CLN2. We are also beginning to work on supplying TPP1 to the rest of the body.  A single injection of stem cells into the eye can produce enough TPP1 to preserve function and structure of the retina for 7 months or longer take cells from the bone marrow Modify the cells to produce TPP1 Inject the cells into the eye and brain where they continue to produce TPP1 long term 1 2 3 Stem Cell Delivery of TPP1 Acknowledgements: Our thanks to collaborators on these projects including Joan Coates, Jacqueline Pearce, Juri Ota-Kuroki, Jeffrey Bryan, Fred Wininger, Dawna Voelkl, Dietrich Volkmann; to the Veterinary Postdocs Stefanie Lim, Katherine Bibi, Daniella Vansteenkiste, Baye Williamson, Whitney Young; to Lani Castaner for assistance with all aspects of the project and to the many students and dogs who made essential contributions to these studies. Funding for these studies was provided by Noah’s Hope/Hope 4 Bridget, Drew's Hope, NIH National Eye Institute, Knights Templar Eye Foundation, and Washington University ICTS. TPP1-expressing cells survived in the brain for 7 months after implantation and were still expressing a marker protein Dogs that had TPP1-expressing cells injected into the cerebrospinal fluid retained cognitive ability longer than untreated dogs Treatment with TPP1-expressing cells prevented retina detachment lesions Untreated Treated (Measureofretinafunction)  Vision loss in CLN2 results from degeneration of both the retina and the brain, so vision preservation will require treatment of both tissues.  When lifespan was extended in dogs that received brain treatment, disease became apparent in other organs outside of the nervous system, such as heart and liver. A cure for CLN2 disease will require treatment for the whole body.