Prenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type III spinal muscular atrophy in mice

Abstract Spinal muscular atrophy (SMA) is a single gene disorder affecting motor function in uterus. Amniotic fluid is an alternative source of stem cell to ameliorate SMA. Therefore, this study aims to examine the therapeutic potential of Human amniotic fluid stem cell (hAFSC) for SMA. Our SMA mode...

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Autores principales:	Steven W. Shaw, Shao-Yu Peng, Ching-Chung Liang, Tzu-Yi Lin, Po-Jen Cheng, T’sang-T’ang Hsieh, Hao-Yu Chuang, Paolo De Coppi, Anna L. David
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/836fd83ebb554beea76411f09fd719db
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spelling	oai:doaj.org-article:836fd83ebb554beea76411f09fd719db2021-12-02T17:39:32ZPrenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type III spinal muscular atrophy in mice10.1038/s41598-021-88559-z2045-2322https://doaj.org/article/836fd83ebb554beea76411f09fd719db2021-04-01T00:00:00Zhttps://doi.org/10.1038/s41598-021-88559-zhttps://doaj.org/toc/2045-2322Abstract Spinal muscular atrophy (SMA) is a single gene disorder affecting motor function in uterus. Amniotic fluid is an alternative source of stem cell to ameliorate SMA. Therefore, this study aims to examine the therapeutic potential of Human amniotic fluid stem cell (hAFSC) for SMA. Our SMA model mice were generated by deletion of exon 7 of Smn gene and knock-in of human SMN2. A total of 16 SMA model mice were injected with 1 × 105 hAFSC in uterus, and the other 16 mice served as the negative control. Motor function was analyzed by three behavioral tests. Engraftment of hAFSC in organs were assessed by flow cytometry and RNA scope. Frequency of myocytes, neurons and innervated receptors were estimated by staining. With hAFSC transplantation, 15 fetuses survived (93.75% survival) and showed better performance in all motor function tests. Higher engraftment frequency were observed in muscle and liver. Besides, the muscle with hAFSC transplantation expressed much laminin α and PAX-7. Significantly higher frequency of myocytes, neurons and innervated receptors were observed. In our study, hAFSC engrafted on neuromuscular organs and improved cellular and behavioral outcomes of SMA model mice. This fetal therapy could preserve the time window and treat in the uterus.Steven W. ShawShao-Yu PengChing-Chung LiangTzu-Yi LinPo-Jen ChengT’sang-T’ang HsiehHao-Yu ChuangPaolo De CoppiAnna L. DavidNature PortfolioarticleMedicineRScienceQENScientific Reports, Vol 11, Iss 1, Pp 1-12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Steven W. Shaw Shao-Yu Peng Ching-Chung Liang Tzu-Yi Lin Po-Jen Cheng T’sang-T’ang Hsieh Hao-Yu Chuang Paolo De Coppi Anna L. David Prenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type III spinal muscular atrophy in mice
description	Abstract Spinal muscular atrophy (SMA) is a single gene disorder affecting motor function in uterus. Amniotic fluid is an alternative source of stem cell to ameliorate SMA. Therefore, this study aims to examine the therapeutic potential of Human amniotic fluid stem cell (hAFSC) for SMA. Our SMA model mice were generated by deletion of exon 7 of Smn gene and knock-in of human SMN2. A total of 16 SMA model mice were injected with 1 × 105 hAFSC in uterus, and the other 16 mice served as the negative control. Motor function was analyzed by three behavioral tests. Engraftment of hAFSC in organs were assessed by flow cytometry and RNA scope. Frequency of myocytes, neurons and innervated receptors were estimated by staining. With hAFSC transplantation, 15 fetuses survived (93.75% survival) and showed better performance in all motor function tests. Higher engraftment frequency were observed in muscle and liver. Besides, the muscle with hAFSC transplantation expressed much laminin α and PAX-7. Significantly higher frequency of myocytes, neurons and innervated receptors were observed. In our study, hAFSC engrafted on neuromuscular organs and improved cellular and behavioral outcomes of SMA model mice. This fetal therapy could preserve the time window and treat in the uterus.
format	article
author	Steven W. Shaw Shao-Yu Peng Ching-Chung Liang Tzu-Yi Lin Po-Jen Cheng T’sang-T’ang Hsieh Hao-Yu Chuang Paolo De Coppi Anna L. David
author_facet	Steven W. Shaw Shao-Yu Peng Ching-Chung Liang Tzu-Yi Lin Po-Jen Cheng T’sang-T’ang Hsieh Hao-Yu Chuang Paolo De Coppi Anna L. David
author_sort	Steven W. Shaw
title	Prenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type III spinal muscular atrophy in mice
title_short	Prenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type III spinal muscular atrophy in mice
title_full	Prenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type III spinal muscular atrophy in mice
title_fullStr	Prenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type III spinal muscular atrophy in mice
title_full_unstemmed	Prenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type III spinal muscular atrophy in mice
title_sort	prenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type iii spinal muscular atrophy in mice
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/836fd83ebb554beea76411f09fd719db
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Prenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type III spinal muscular atrophy in mice

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