Article
CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2016
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
Abstract excerpt
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 Nucleases (TALENs) and the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 nuclease system can...
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