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2016 BDSRA Whiting & Katz CLN2

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Long-term delivery of TPP1 enzyme to the eye and brain in CLN2

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2016 BDSRA Whiting & Katz CLN2

  1. 1. CLN2 Long-term delivery of TPP1 enzyme to the eye and brain in CLN2 Rebecca Whiting, Martin Katz, Ophthalmology, School of Medicine, University of Missouri Acknowledgements: Our thanks to collaborators on these projects including Joan Coates, Jacqueline Pearce, Jeffrey Bryan, Fred Wininger, Dawna Voekl, Dietrich Voelkman; to the Veterinary Post-docs 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. Financial support for these projects was provided by the National Institutes of Health and the Knights Templar Eye Foundation. INTRODUCTION  CLN2 disease is caused by a lack of TPP1 enzyme, and supplying the enzyme to the brain delays symptom progression  Our laboratory is working to optimize long-term delivery of the enzyme to the brain and eye using a dog model of CLN2 0 1 2 3 4 5 6 7 8 9 Normal Treated, CLN2 Untreated, CLN2 #ofIncorrectChoices Measure of Learning Ability Treated, CLN2 Untreated, CLN2 TREATMENT PRESERVES VISUAL FUNCTION TREATMENT PRESERVES COGNITIVE FUNCTION 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 TREATED UNTREATED 0 2 4 6 8 10 12 Normal Treated, CLN2 Untreated, CLN2 #ofIncorrectChoices Measure of Learning Ability METHODS #ErrorsonCognitiveTest Retina response to flash of light Treatment prevents retina lesions CONCLUSIONS  A single injection of stem cells into the eye can produce enough TPP1 to preserve visual function for 7 months or longer  Preliminary results suggest that treatment with the same cells will preserve the brain long term  We are making progress towards a one-time curative treatment for CLN2 disease.

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