Nature Communications (Sep 2017)
Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
- Sachith Mettananda,
- Chris A. Fisher,
- Deborah Hay,
- Mohsin Badat,
- Lynn Quek,
- Kevin Clark,
- Philip Hublitz,
- Damien Downes,
- Jon Kerry,
- Matthew Gosden,
- Jelena Telenius,
- Jackie A. Sloane-Stanley,
- Paula Faustino,
- Andreia Coelho,
- Jessica Doondeea,
- Batchimeg Usukhbayar,
- Paul Sopp,
- Jacqueline A. Sharpe,
- Jim R. Hughes,
- Paresh Vyas,
- Richard J. Gibbons,
- Douglas R. Higgs
Affiliations
- Sachith Mettananda
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Chris A. Fisher
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Deborah Hay
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Mohsin Badat
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Lynn Quek
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Kevin Clark
- MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Philip Hublitz
- MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Damien Downes
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Jon Kerry
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Matthew Gosden
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Jelena Telenius
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Jackie A. Sloane-Stanley
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Paula Faustino
- Human Genetics Department, National Institute of Health Dr. Ricardo Jorge, Av. Padre Cruz
- Andreia Coelho
- Human Genetics Department, National Institute of Health Dr. Ricardo Jorge, Av. Padre Cruz
- Jessica Doondeea
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Batchimeg Usukhbayar
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Paul Sopp
- MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Jacqueline A. Sharpe
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Jim R. Hughes
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Paresh Vyas
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Richard J. Gibbons
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- Douglas R. Higgs
- Medical Research Council (MRC) Molecular Hematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford
- DOI
- https://doi.org/10.1038/s41467-017-00479-7
- Journal volume & issue
-
Vol. 8,
no. 1
pp. 1 – 11
Abstract
β-thalassemia is characterised by the presence of an excess of α-globin chains, which contribute to erythrocyte pathology. Here the authors use CRISP/Cas9 to reduce α-globin expression in hematopoietic precursors, and show effectiveness in xenograft assays in mice.
