Article
Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.
Blood - 23 Apr 2015
Hoban Megan D, Cost Gregory J, Mendel Matthew C, Romero Zulema, Kaufman Michael L, Joglekar Alok V, Ho Michelle, Lumaquin Dianne, Gray David, Lill Georgia R, Cooper Aaron R, Urbinati Fabrizia, Senadheera Shantha, Zhu Allen, Liu Pei-Qi, Paschon David E, Zhang Lei, Rebar Edward J, Wilber Andrew, Wang Xiaoyan, Gregory Philip D, Holmes Michael C, Reik Andreas, Hollis Roger P, Kohn Donald B
Abstract excerpt
Sickle cell disease (SCD) is characterized by a single point mutation in the seventh codon of the β-globin gene. Site-specific correction of the sickle mutation in hematopoietic stem cells would allow for permanent production of normal red blood cells. Using zinc-finger nucleases (ZFNs) designed to flank the sickle mutation, we demonstrate efficient targeted cleavage at the β-globin locus with minimal off-target...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.