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 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...

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Detalles Bibliográficos
Autores principales: Tian, Guoshuai, Cao, Chunyu, Li, Shuyue, Wang, Wei, Zhang, Ye, Lv, Yafeng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10143348/
https://www.ncbi.nlm.nih.gov/pubmed/37111381
http://dx.doi.org/10.3390/ph16040624

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10143348/
https://www.ncbi.nlm.nih.gov/pubmed/37111381
http://dx.doi.org/10.3390/ph16040624

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