CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells

Targeted genome editing technology can correct the sickle cell disease mutation of the β-globin gene in hematopoietic stem cells. This correction supports production of red blood cells that synthesize normal hemoglobin proteins. Here, we demonstrate that Transcription Activator-Like Effector Nucleas...

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Publicat a:Mol Ther
Autors principals: Hoban, Megan D, Lumaquin, Dianne, Kuo, Caroline Y, Romero, Zulema, Long, Joseph, Ho, Michelle, Young, Courtney S, Mojadidi, Michelle, Fitz-Gibbon, Sorel, Cooper, Aaron R, Lill, Georgia R, Urbinati, Fabrizia, Campo-Fernandez, Beatriz, Bjurstrom, Carmen F, Pellegrini, Matteo, Hollis, Roger P, Kohn, Donald B
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2016
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5113113/
https://ncbi.nlm.nih.gov/pubmed/27406980
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.148
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