Induced neural stem cells from human patient-derived fibroblasts attenuate neurodegeneration in Niemann-Pick type C mice.

ABSTRACT:

Background

Niemann-Pick disease type C (NPC) is caused by the mutation of NPC genes, which leads to the abnormal accumulation of unesterified cholesterol and glycolipids in lysosomes. This autosomal recessive disease is characterized by liver dysfunction, hepatosplenomegaly, and progressive neurodegeneration. Recently, the application of induced neural stem cells (iNSCs), converted from fibroblasts using specific transcription factors, to repair degenerated lesions has been considered a novel therapy.

Objectives

The therapeutic effects on NPC by human iNSCs generated by our research group have not yet been studied in vivo; in this study, we investigate those effects.

Methods

We used an NPC mouse model to efficiently evaluate the therapeutic effect of iNS

SUBMITTER: Hong S 

PROVIDER: S-EPMC7850792 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature