Journal
PLOS ONE
Volume 6, Issue 3, Pages -Publisher
PUBLIC LIBRARY SCIENCE
DOI: 10.1371/journal.pone.0017565
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Categories
Funding
- European Program 7FWP, Health [222878]
- Chronic Granulomatous Disorder Research Trust [J4G/04B/GT09]
- Biotechnology and Biological Sciences Research Council, UK [BB/F015526/1]
- Wellcome Trust [090233/Z/09/Z]
- GOSH Children's Charity
- Ministry of Science and Innovation for Programa de Fomento de Cooperacion Cientifica Internacional [110-90.1]
- Plan Nacional de Salud y Farmacia [SAF 2009-07164]
- Fondo de Investigaciones Sanitarias, ISCIII [RETICS-RD06/0010/0015]
- Wellcome Trust [090233/Z/09/Z] Funding Source: Wellcome Trust
- BBSRC [BB/F015526/1] Funding Source: UKRI
- Biotechnology and Biological Sciences Research Council [BB/F015526/1] Funding Source: researchfish
- Great Ormond Street Hospital Childrens Charity [V1223] Funding Source: researchfish
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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.
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