Genetic Correction of Concurrent α- and β-Thalassemia Patient-Derived Pluripotent Stem Cells by the CRISPR-Cas9 Technology

The authors established a new hiPSCs line derived from amniotic cells of a fetus with a homozygous β41-42 deletion mutation in the HBB gene and a heterozygous Westmead mutation in the HBA2 gene. They designed a CRISPR-Cas9 to target these casual mutations and corrected them.