Gene correction of HBB mutations in CD34+ hematopoietic stem cells using Cas9 mRNA and ssODN donors.
Justin S AntonyNgadhnjim LatifiA K M Ashiqul HaqueAndrés Lamsfus-CalleAlberto Daniel-MorenoSebastian GraeterPraveen BaskaranPetra WeinmannMarkus MezgerRupert HandgretingerMichael S D KormannPublished in: Molecular and cellular pediatrics (2018)
Our approach provides guidance on non-viral gene correction in CD34+ HSCs using Cas9 mRNA and chemically modified ssODN. However, further optimization is needed to increase the homology directed repair (HDR) to attain a real clinical benefit for β-thalassemia.