rAAV2-Mediated Restoration of GALC in Neural Stem Cells from Krabbe Patient-Derived iPSCs

rAAV2-Mediated Restoration of GALC in Neural Stem Cells from Krabbe Patient-Derived iPSCs

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Krabbe disease is a rare neurodegenerative fatal disease.It is caused by deficiency of the lysosomal enzyme galactocerebrosidase (GALC), which results in pet calming peanut butter 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 generated induced pluripotent stem cells (iPSCs) from a Krabbe patient previously.Here, Krabbe patient-derived neural stem cells (K-NSCs) were induced from these iPSCs.By using nine kinds of recombinant adeno-associated virus (rAAV) vectors to infect K-NSCs, we found that the rAAV2 vector has high transduction efficiency for K-NSCs.

Most importantly, rAAV2-GALC rescued GALC enzymatic activity in K-NSCs.Our findings not only establish a novel patient NSC model for Krabbe disease, read more but also firstly indicate the potential of rAAV2-mediated gene therapy for this devastating disease.