rAAV2-Mediated Restoration of GALC in Neural Stem Cells from Krabbe Patient-Derived iPSCs
Krabbe disease is a rare neurodegenerative fatal disease.It is caused by Cat Beds deficiency of the lysosomal enzyme galactocerebrosidase (GALC), which results in progressive accumulation of galactolipid substrates in myelin-forming cells.However, there is still a lack of appropriate neural models and effective approaches for Krabbe disease.We gene