CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells.
Jennifer E. Chung, Wendy Magis, Jonathan T. Vu, Seok‐Jin Heo, Kirmo Wartiovaara, Mark C. Walters, Ryo Kurita, Yukio Nakamura, Dario Boffelli, David I. K. Martin, Jacob E. Corn, Mark A. DeWitt
This study uses CRISPR-Cas9 to test a putative fetal globin repressor region, finding it does not autonomously silence gamma-globin, suggesting it is not a therapeutic target for beta-hemoglobinopathies.
PLoS ONE · Jan 15, 2019Peer-reviewedGenetics