Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells

By Á. Raya, Ignasi Rodriguez-Piza, G. Guenechea, R. Vassena, S. Navarro, M. J. Barrero, A. Consiglio, M. Castellà, P. Río, Eduard Sleep and 9 more. Nature, 2009

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The generation of induced pluripotent stem (iPS) cells has enabled the derivation of patient-specific pluripotent cells and provided valuable experimental platforms to model human disease. Patient-specific iPS cells are also thought to hold great therapeutic potential, although direct evidence for this is still lacking. Here we show that, on correction of the genetic defect, somatic cells from Fanconi anaemia patients can be reprogrammed to pluripotency to generate patient-specific iPS cells. These cell lines appear indistinguishable from human embryonic stem cells and iPS cells from healthy individuals. Most importantly, we show that corrected Fanconi-anaemia-specific iPS cells can give rise to haematopoietic progenitors of the myeloid and erythroid lineages that are phenotypically normal, that is, disease-free. These data offer proof-of-concept that iPS cell technology can be used for the generation of disease-corrected, patient-specific cells with potential value for cell therapy applications.

Subfields: Molecular Biology

View Paper: https://www.semanticscholar.org/paper/569f759b7933ad682f5fdbe01e28ba5e75d23508

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Citations in the corpus

• Induced pluripotency: history, mechanisms, and applications. (2010, background): “…GENES & DEVELOPMENT iPSCs from patients suffering from Huntington’s and Parkinson’s disease, ALS, juvenile diabetes, muscular dystrophy, Fanconi anemia, Down syndrome, and others (Dimos et al. 2008; Park et al. 2008a; Raya et al. 2009; Soldner et al. 2009), which will facilitate these studies.”
• Gene therapy clinical trials worldwide to 2012 – an update (2013, background): “Gene-corrected iPS cells derived from Fanconi anaemia patients have also been differentiated into haematopoietic progenitors of the myeloid and erythroid lineages [84] and may be useful for overcoming the poor quality of HSCs found in the bone marrow of these patients, which is impeding success in t”