Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia

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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Autores principales: 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
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2017
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Science
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Acceso en línea:https://doaj.org/article/0cd23b9b09ec4af18f7d483a67ceba18
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https://doaj.org/article/0cd23b9b09ec4af18f7d483a67ceba18

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