Xenografting of human umbilical mesenchymal stem cells from Wharton's jelly ameliorates mouse spinocerebellar ataxia type 1.

ABSTRACT:

Background

Spinocerebellar ataxia type 1 (SCA1) is an autosomal dominant neurodegenerative disorder caused by the expansion of CAG repeats in ATXN1 gene resulting in an expansion of polyglutamine repeats in the ATXN1 protein. Unfortunately, there has yet been any effective treatment so far for SCA1. This study investigated the feasibility of transplanting human umbilical mesenchymal stem cells (HUMSCs) into transgenic SCA1 mice containing an expanded uninterrupted allele with 82 repeats in the ATXN1-coding region.

Methods

10⁶ human umbilical mesenchymal stem cells were transplanted into the cerebella at 1 month of age.

Results

HUMSCs displayed significant ameliorating effects in SCA1 mice in terms of motor behaviors in balance beam test and op

SUBMITTER: Tsai PJ 

PROVIDER: S-EPMC6727337 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature