Genetically corrected iPSC-derived Neural Stem Cell Grafts deliver NAGLU-IGFII fusion protein to affect CNS disease in Sanfilippo B Mice
Yewande Pearse, Don Clarke, Shih‐hsin Kan, Steven Q. Le, Valentina Sanghez, Anna Luzzi, Ivy Pham, Lina R. Nih, Jonathan D. Cooper, Patricia Dickson, Michelina Iacovino
This study genetically corrects iPSC-derived neural stem cells to deliver a modified NAGLU enzyme, reducing disease pathology in Sanfilippo B mice.
bioRxiv · Jun 30, 2022 · PreprintNeuroscience