The DOI matches its Crossref record; 4 of the 4 references it cites were found in a registry or an index.
Treatment of Sickle Cell Anemia Mouse Model with iPS Cells Generated from Autologous Skin
Abstract
It has recently been demonstrated that mouse and human fibroblasts can be reprogrammed into an embryonic stem cell–like state by introducing combinations of four transcription factors. However, the therapeutic potential of such induced pluripotent stem (iPS) cells remained undefined. By using a humanized sickle cell anemia mouse model, we show that mice can be rescued after transplantation with hematopoietic progenitors obtained in vitro from autologous iPS cells. This was achieved after correction of the human sickle hemoglobin allele by gene-specific targeting. Our results provide proof of principle for using transcription factor–induced reprogramming combined with gene and cell therapy for disease treatment in mice. The problems associated with using retroviruses and oncogenes for reprogramming need to be resolved before iPS cells can be considered for human therapy.
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“A recent paper by Hanna et al. establishes prove-of- principal for the correction of an inherited blood disorder with this approach by demonstrating the derivation of engraftable hematopoietic stem cells generated in vitro from autologous iPS cells in a humanized sickle cell anemia mouse model[104].”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [104]
Citations
Cites
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Pathogenesis and Treatment of Sickle Cell Disease
Bunn, H. F. (1997) · New England Journal of Medicine · doi:10.1056/nejm199709113371107 · Cited by 1,154
Correction of sickle cell disease in transgenic mouse models by gene therapy
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Cited by
Rees, D. C., Williams, T. N., & Gladwin, M. T. (2010) · The Lancet · doi:10.1016/s0140-6736(10)61029-x · Cited by 2,504
Generation of Human Induced Pluripotent Stem Cells by Direct Delivery of Reprogramming Proteins
Kim, D., Kim, C.-H., Moon, J.-I., Chung, Y.-G., Chang, M.-Y., Han, B.-S., Ko, S., Yang, E., Cha, K. Y., Lanza, R., et al. (2009) · Cell stem cell · doi:10.1016/j.stem.2009.05.005 · Cited by 1,814
Induced pluripotent stem cells generated without viral integration
Stadtfeld, M., Nagaya, M., Utikal, J., Weir, G., & Hochedlinger, K. (2008) · Science · doi:10.1126/science.1162494 · Cited by 1,683
Induced pluripotent stem cell technology: a decade of progress.
Shi, Y., Inoue, H., Wu, J. C., & Yamanaka, S. (2017) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2016.245 · Cited by 1,525
Advances in organ-on-a-chip engineering
Zhang, B., Korolj, A., Lai, B. F. L., & Radisic, M. (2018) · Nature Reviews Materials · doi:10.1038/s41578-018-0034-7 · Cited by 1,017
Metadata
- Authors
- Hanna, J., Wernig, M., Markoulaki, S., Sun, C.-W., Meissner, A., Cassady, J. P., Beard, C., Brambrink, T., Wu, L.-C., Townes, T. M., & Jaenisch, R.
- Affiliations
- The Whitehead Institute for Biomedical Research, Cambridge, MA 02142, USA.
- Venue
- Science
- Published
- 2007-12-21
- Volume
- 318
- Issue
- 5858
- Pages
- 1920-1923
- ISSN
- 0036-8075
- 1095-9203
- Cited by
- 1,202
- References
- 4
- Language
- en
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