Stem Cell Delivery of TTP1 in Batten Disease Dogs – Rare Disease Report
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Stem Cell Delivery of TTP1 in Batten Disease Dogs
Rare Disease Report 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 … |
