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

Pei Jiun Tsai, Chang Ching Yeh, Wan Jhen Huang, Ming Yuan Min, Tzu Hao Huang, Tsui Ling Ko, Pei Yu Huang, Tien Hua Chen, Sanford P.C. Hsu, Bing Wen Soong, Yu Show Fu

Research output: Contribution to journalArticlepeer-review

11 Citations (Scopus)


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 open field test as compared with the untransplanted SCA1 mice. HUMSCs transplantation effectively reduced the cerebellar atrophy, salvaged Purkinje cell death, and alleviated molecular layer shrinkage. Electrophysiological studies showed higher amplitudes of compound motor action potentials as indicated by increasing neuronal-muscular response strength to stimuli after stem cell transplantation. At 5 months after transplantation, HUMSCs scattering in the mice cerebella remained viable and secreted cytokines without differentiating into neuronal or glia cells. Conclusions: Our findings provide hope for a new therapeutic direction for the treatment of SCA1.

Original languageEnglish
Article number29
JournalTranslational Neurodegeneration
Issue number1
Publication statusPublished - Sep 5 2019


  • Cell transplantation
  • SCA1
  • Umbilical mesenchymal stem cells

ASJC Scopus subject areas

  • Clinical Neurology
  • Cognitive Neuroscience
  • Cellular and Molecular Neuroscience


Dive into the research topics of 'Xenografting of human umbilical mesenchymal stem cells from Wharton's jelly ameliorates mouse spinocerebellar ataxia type 1'. Together they form a unique fingerprint.

Cite this