NeurosciencebioRxivPreprint
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…·Michelina Iacovino
This study genetically corrects iPSC-derived neural stem cells to deliver a modified NAGLU enzyme, reducing disease pathology in Sanfilippo B mice.
DOI 10.1101/2022.06.30.498131