Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9 Enhanced Gene Replacement, Homozygous correction
To determine the specificity of CRISPR/Cas9 directed JAK3 correction in human SCID iPSC, whole-genome sequencing was applied before and after gene replacement. One parental iPSC line, two heterozygous corrected line and one homozygous corrected line were used for WGS.