Article
Modeling Sialidosis with Neural Precursor Cells Derived from Patient-Derived Induced Pluripotent Stem Cells.
International journal of molecular sciences - 22 Apr 2021
Seol Binna, Kim Young-Dae, Cho Yee Sook
Abstract excerpt
Sialidosis, caused by a genetic deficiency of the lysosomal sialidase gene (NEU1), is a systemic disease involving various tissues and organs, including the nervous system. Understanding the neurological dysfunction and pathology associated with sialidosis remains a challenge, partially due to the lack of a human model system. In this study, we have generated two types of induced pluripotent stem cells (iPSCs)...
Topics
- Astrocytes
- Autophagy
- Cell Differentiation
- Humans
- Induced Pluripotent Stem Cells
- Lysosomes
- Mucolipidoses
- Mutation
- Neural Stem Cells
- Neuraminidase
- Oligodendroglia
