Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy

Confocal picture showing the myelinogenic potential of hiPS-NSCs engrafted at the border between the striatum and the corpus callosum of immunodeficient MLD mice at 3 months post-transplantation(green, human cells labeled with anti-human mitochondria; red, myelin basic protein; blue, nuclei counterstained with ToPro3). Scale bar: 100µm.