Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.

Murine models of human genetic disorders provide a valuable tool for investigating the scope for application of induced pluripotent stem cells (iPSC). Here we present a proof-of-concept study to demonstrate generation of iPSC from a mouse model of X-linked chronic granulomatous disease (X-CGD), and...

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Autores principales:	Sayandip Mukherjee, Giorgia Santilli, Michael P Blundell, Susana Navarro, Juan A Bueren, Adrian J Thrasher
Formato:	article
Lenguaje:	EN
Publicado:	Public Library of Science (PLoS) 2011
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/466dc23b398e4aa3b5f0e72bccaad3d1
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spelling	oai:doaj.org-article:466dc23b398e4aa3b5f0e72bccaad3d12021-11-18T06:57:48ZGeneration of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.1932-620310.1371/journal.pone.0017565https://doaj.org/article/466dc23b398e4aa3b5f0e72bccaad3d12011-03-01T00:00:00Zhttps://www.ncbi.nlm.nih.gov/pmc/articles/pmid/21408614/?tool=EBIhttps://doaj.org/toc/1932-6203Murine models of human genetic disorders provide a valuable tool for investigating the scope for application of induced pluripotent stem cells (iPSC). Here we present a proof-of-concept study to demonstrate generation of iPSC from a mouse model of X-linked chronic granulomatous disease (X-CGD), and their successful differentiation into haematopoietic progenitors of the myeloid lineage. We further demonstrate that additive gene transfer using lentiviral vectors encoding gp91(phox) is capable of restoring NADPH-oxidase activity in mature neutrophils derived from X-CGD iPSC. In the longer term, correction of iPSC from human patients with CGD has therapeutic potential not only through generation of transplantable haematopoietic stem cells, but also through production of large numbers of autologous functional neutrophils.Sayandip MukherjeeGiorgia SantilliMichael P BlundellSusana NavarroJuan A BuerenAdrian J ThrasherPublic Library of Science (PLoS)articleMedicineRScienceQENPLoS ONE, Vol 6, Iss 3, p e17565 (2011)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Sayandip Mukherjee Giorgia Santilli Michael P Blundell Susana Navarro Juan A Bueren Adrian J Thrasher Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.
description	Murine models of human genetic disorders provide a valuable tool for investigating the scope for application of induced pluripotent stem cells (iPSC). Here we present a proof-of-concept study to demonstrate generation of iPSC from a mouse model of X-linked chronic granulomatous disease (X-CGD), and their successful differentiation into haematopoietic progenitors of the myeloid lineage. We further demonstrate that additive gene transfer using lentiviral vectors encoding gp91(phox) is capable of restoring NADPH-oxidase activity in mature neutrophils derived from X-CGD iPSC. In the longer term, correction of iPSC from human patients with CGD has therapeutic potential not only through generation of transplantable haematopoietic stem cells, but also through production of large numbers of autologous functional neutrophils.
format	article
author	Sayandip Mukherjee Giorgia Santilli Michael P Blundell Susana Navarro Juan A Bueren Adrian J Thrasher
author_facet	Sayandip Mukherjee Giorgia Santilli Michael P Blundell Susana Navarro Juan A Bueren Adrian J Thrasher
author_sort	Sayandip Mukherjee
title	Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.
title_short	Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.
title_full	Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.
title_fullStr	Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.
title_full_unstemmed	Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.
title_sort	generation of functional neutrophils from a mouse model of x-linked chronic granulomatous disorder using induced pluripotent stem cells.
publisher	Public Library of Science (PLoS)
publishDate	2011
url	https://doaj.org/article/466dc23b398e4aa3b5f0e72bccaad3d1
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Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.

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