Prenatal transplantation of mesenchymal stem cells to treat osteogenesis imperfecta

Osteogenesis imperfecta (OI) can be a severe disorder that can be diagnosed before birth. Transplantation of mesenchymal stem cells (MSC) has the potential to improve the bone structure, growth, and fracture healing. In this review, we give an introduction to OI and MSC, and the basis for pre- and p...

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Main Authors:	Chan, Jerry K. Y., Götherström, Cecilia
Format:	Online
Language:	English
Published:	Frontiers Media S.A. 2014
Online Access:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4191163/

id	pubmed-4191163
recordtype	oai_dc
spelling	pubmed-41911632014-10-24 Prenatal transplantation of mesenchymal stem cells to treat osteogenesis imperfecta Chan, Jerry K. Y. Götherström, Cecilia Pharmacology Osteogenesis imperfecta (OI) can be a severe disorder that can be diagnosed before birth. Transplantation of mesenchymal stem cells (MSC) has the potential to improve the bone structure, growth, and fracture healing. In this review, we give an introduction to OI and MSC, and the basis for pre- and postnatal transplantation in OI. We also summarize the two patients with OI who have received pre- and postnatal transplantation of MSC. The findings suggest that prenatal transplantation of allogeneic MSC in OI is safe. The cell therapy is of likely clinical benefit with improved linear growth, mobility, and reduced fracture incidence. Unfortunately, the effect is transient. For this reason, postnatal booster infusions using same-donor MSC have been performed with clinical benefit, and without any adverse events. So far there is limited experience in this specific field and proper studies are required to accurately conclude on clinical benefits of MSC transplantation to treat OI. Frontiers Media S.A. 2014-10-09 /pmc/articles/PMC4191163/ /pubmed/25346689 http://dx.doi.org/10.3389/fphar.2014.00223 Text en Copyright © 2014 Chan and Götherström. http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
repository_type	Open Access Journal
institution_category	Foreign Institution
institution	US National Center for Biotechnology Information
building	NCBI PubMed
collection	Online Access
language	English
format	Online
author	Chan, Jerry K. Y. Götherström, Cecilia
spellingShingle	Chan, Jerry K. Y. Götherström, Cecilia Prenatal transplantation of mesenchymal stem cells to treat osteogenesis imperfecta
author_facet	Chan, Jerry K. Y. Götherström, Cecilia
author_sort	Chan, Jerry K. Y.
title	Prenatal transplantation of mesenchymal stem cells to treat osteogenesis imperfecta
title_short	Prenatal transplantation of mesenchymal stem cells to treat osteogenesis imperfecta
title_full	Prenatal transplantation of mesenchymal stem cells to treat osteogenesis imperfecta
title_fullStr	Prenatal transplantation of mesenchymal stem cells to treat osteogenesis imperfecta
title_full_unstemmed	Prenatal transplantation of mesenchymal stem cells to treat osteogenesis imperfecta
title_sort	prenatal transplantation of mesenchymal stem cells to treat osteogenesis imperfecta
description	Osteogenesis imperfecta (OI) can be a severe disorder that can be diagnosed before birth. Transplantation of mesenchymal stem cells (MSC) has the potential to improve the bone structure, growth, and fracture healing. In this review, we give an introduction to OI and MSC, and the basis for pre- and postnatal transplantation in OI. We also summarize the two patients with OI who have received pre- and postnatal transplantation of MSC. The findings suggest that prenatal transplantation of allogeneic MSC in OI is safe. The cell therapy is of likely clinical benefit with improved linear growth, mobility, and reduced fracture incidence. Unfortunately, the effect is transient. For this reason, postnatal booster infusions using same-donor MSC have been performed with clinical benefit, and without any adverse events. So far there is limited experience in this specific field and proper studies are required to accurately conclude on clinical benefits of MSC transplantation to treat OI.
publisher	Frontiers Media S.A.
publishDate	2014
url	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4191163/
_version_	1613142471098761216

Prenatal transplantation of mesenchymal stem cells to treat osteogenesis imperfecta

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