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

• Main Authors: Guoshuai Tian (Author), Chunyu Cao (Author), Shuyue Li (Author), Wei Wang (Author), Ye Zhang (Author), Yafeng Lv (Author)
• Formato: Livro
• Publicado em: MDPI AG, 2023-04-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

Krabbe disease is a rare neurodegenerative fatal disease. It is caused by 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 effe...