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In utero hematopoietic cell transplantation for hemoglobinopathies

In utero hematopoietic cell transplantation (IUHCTx) is a promising strategy to circumvent the challenges of postnatal hematopoietic stem cell (HSC) transplantation. The goal of IUHCTx is to introduce donor cells into a naïve host prior to immune maturation, thereby inducing donor–specific tolerance...

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Autores principales: Derderian, S. Christopher, Jeanty, Cerine, Walters, Mark C., Vichinsky, Elliott, MacKenzie, Tippi C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4290536/
https://www.ncbi.nlm.nih.gov/pubmed/25628564
http://dx.doi.org/10.3389/fphar.2014.00278
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author Derderian, S. Christopher
Jeanty, Cerine
Walters, Mark C.
Vichinsky, Elliott
MacKenzie, Tippi C.
author_facet Derderian, S. Christopher
Jeanty, Cerine
Walters, Mark C.
Vichinsky, Elliott
MacKenzie, Tippi C.
author_sort Derderian, S. Christopher
collection PubMed
description In utero hematopoietic cell transplantation (IUHCTx) is a promising strategy to circumvent the challenges of postnatal hematopoietic stem cell (HSC) transplantation. The goal of IUHCTx is to introduce donor cells into a naïve host prior to immune maturation, thereby inducing donor–specific tolerance. Thus, this technique has the potential of avoiding host myeloablative conditioning with cytotoxic agents. Over the past two decades, several attempts at IUHCTx have been made to cure numerous underlying congenital anomalies with limited success. In this review, we will briefly review the history of IUHCTx and give a perspective on alpha thalassemia major, one target disease for its clinical application.
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spelling pubmed-42905362015-01-27 In utero hematopoietic cell transplantation for hemoglobinopathies Derderian, S. Christopher Jeanty, Cerine Walters, Mark C. Vichinsky, Elliott MacKenzie, Tippi C. Front Pharmacol Pharmacology In utero hematopoietic cell transplantation (IUHCTx) is a promising strategy to circumvent the challenges of postnatal hematopoietic stem cell (HSC) transplantation. The goal of IUHCTx is to introduce donor cells into a naïve host prior to immune maturation, thereby inducing donor–specific tolerance. Thus, this technique has the potential of avoiding host myeloablative conditioning with cytotoxic agents. Over the past two decades, several attempts at IUHCTx have been made to cure numerous underlying congenital anomalies with limited success. In this review, we will briefly review the history of IUHCTx and give a perspective on alpha thalassemia major, one target disease for its clinical application. Frontiers Media S.A. 2015-01-12 /pmc/articles/PMC4290536/ /pubmed/25628564 http://dx.doi.org/10.3389/fphar.2014.00278 Text en Copyright © 2015 Derderian, Jeanty, Walters, Vichinsky and MacKenzie. 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.
spellingShingle Pharmacology
Derderian, S. Christopher
Jeanty, Cerine
Walters, Mark C.
Vichinsky, Elliott
MacKenzie, Tippi C.
In utero hematopoietic cell transplantation for hemoglobinopathies
title In utero hematopoietic cell transplantation for hemoglobinopathies
title_full In utero hematopoietic cell transplantation for hemoglobinopathies
title_fullStr In utero hematopoietic cell transplantation for hemoglobinopathies
title_full_unstemmed In utero hematopoietic cell transplantation for hemoglobinopathies
title_short In utero hematopoietic cell transplantation for hemoglobinopathies
title_sort in utero hematopoietic cell transplantation for hemoglobinopathies
topic Pharmacology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4290536/
https://www.ncbi.nlm.nih.gov/pubmed/25628564
http://dx.doi.org/10.3389/fphar.2014.00278
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