Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
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.
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Nature Portfolio
2017
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oai:doaj.org-article:0cd23b9b09ec4af18f7d483a67ceba182021-12-02T15:38:49ZEditing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia10.1038/s41467-017-00479-72041-1723https://doaj.org/article/0cd23b9b09ec4af18f7d483a67ceba182017-09-01T00:00:00Zhttps://doi.org/10.1038/s41467-017-00479-7https://doaj.org/toc/2041-1723β-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.Sachith MettanandaChris A. FisherDeborah HayMohsin BadatLynn QuekKevin ClarkPhilip HublitzDamien DownesJon KerryMatthew GosdenJelena TeleniusJackie A. Sloane-StanleyPaula FaustinoAndreia CoelhoJessica DoondeeaBatchimeg UsukhbayarPaul SoppJacqueline A. SharpeJim R. HughesParesh VyasRichard J. GibbonsDouglas R. HiggsNature PortfolioarticleScienceQENNature Communications, Vol 8, Iss 1, Pp 1-11 (2017) |
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DOAJ |
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DOAJ |
| language |
EN |
| topic |
Science Q |
| spellingShingle |
Science Q 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 Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia |
| description |
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. |
| format |
article |
| author |
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 |
| author_facet |
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 |
| author_sort |
Sachith Mettananda |
| title |
Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia |
| title_short |
Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia |
| title_full |
Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia |
| title_fullStr |
Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia |
| title_full_unstemmed |
Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia |
| title_sort |
editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia |
| publisher |
Nature Portfolio |
| publishDate |
2017 |
| url |
https://doaj.org/article/0cd23b9b09ec4af18f7d483a67ceba18 |
| work_keys_str_mv |
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1718386033055236096 |