Naïve iPSCs Generated from β-thalassemia Fibroblasts Allow Efficient Gene Correction with CRISPR/Cas9. Homo sapiens

We used a modified 5i/L/FA system to generate transgene-free naïve iPSCs directly from the fibroblasts of a patient suffering from β-thalassemia and further demonstrated efficient gene correction with a CRISPR/Cas9 system, which provides an improved strategy for personalized treatment of β-thalassemia. Overall design: A modified 5i/L/FA system was used to generate transgene-free naïve iPSCs directly from the fibroblasts of a patient suffering from β-thalassemia.