Figure 4.
Figure 4. Development of SCD patients. (A) Studies deriving gene-corrected HSCs using human cell lines obtained from patients with SCD to generate iPS cells, with the ultimate goal of autologous transplantation. Reprinted with permission from Freed et al, 2012.21 (B) Sleeping Beauty (SB) transposon and transposase mediated wild-type (Wt) β-globin gene integration. (C) Transcription activator-like effector nucleases (TALENs)-mediated human beta-globin (HBB) gene correction in SCD. (D) The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas)-mediated human β-globin (HBB) gene correction in SCD.

Development of SCD patients. (A) Studies deriving gene-corrected HSCs using human cell lines obtained from patients with SCD to generate iPS cells, with the ultimate goal of autologous transplantation. Reprinted with permission from Freed et al, 2012.21  (B) Sleeping Beauty (SB) transposon and transposase mediated wild-type (Wt) β-globin gene integration. (C) Transcription activator-like effector nucleases (TALENs)-mediated human beta-globin (HBB) gene correction in SCD. (D) The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas)-mediated human β-globin (HBB) gene correction in SCD.

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