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

Spinocerebellar ataxia type 1 (SCA1) is an autosomal dominant neurodegenerative disorder caused by the expansion of CAG repeats in 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 inves...

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Published inTranslational neurodegeneration Vol. 8; no. 1; p. 29
Main Authors Tsai, Pei-Jiun, Yeh, Chang-Ching, Huang, Wan-Jhen, Min, Ming-Yuan, Huang, Tzu-Hao, Ko, Tsui-Ling, Huang, Pei-Yu, Chen, Tien-Hua, Hsu, Sanford P C, Soong, Bing-Wen, Fu, Yu-Show
Format Journal Article
LanguageEnglish
Published England BioMed Central 05.09.2019
BMC
Subjects
Age
Ataxia
Cell transplantation
Chromosomes
Cytokines
Experiments
Laboratory animals
Rodents
SCA1
Stem cells
Transgenic animals
Transplants & implants
Umbilical cord
Umbilical mesenchymal stem cells
Umbilical mesenchymal stem cells
SCA1
Cell transplantation
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Summary:Spinocerebellar ataxia type 1 (SCA1) is an autosomal dominant neurodegenerative disorder caused by the expansion of CAG repeats in 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 coding region. 10 human umbilical mesenchymal stem cells were transplanted into the cerebella at 1 month of age. 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. Our findings provide hope for a new therapeutic direction for the treatment of SCA1.
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ISSN:2047-9158
2047-9158
DOI:10.1186/s40035-019-0166-8