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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 Kormann
Published 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.
Keyphrases
  • stem cells
  • crispr cas
  • genome editing
  • copy number
  • genome wide
  • genome wide identification
  • sars cov
  • binding protein
  • bone marrow
  • cell therapy
  • dna methylation
  • mesenchymal stem cells