Figure 5.

Repair of the PiZZ 1024 G > A mutation in induced pluripotent stem cells (iPSCs) derived from a patient with alpha-1-antitrypsin deficiency. (A) Schematic of the disease-associated 1024 G > A mutation in the SERPINA1 gene, the altered protein sequence (E342K), and the sgRNA used to correct the mutation with NG-ABEmax. (B) Frequencies of A-to-G conversions in the editing window induced by NG-ABEmax. (C) Frequences of A-to-G conversions at the G > A mutation site induced by NG-PE3 system. pegRNAs with varying RT lengths were tested. (D) Comparison of the frequencies of precise correction of the PiZZ mutation in patient-derived iPSCs by NG-PE3 and NG-ABEmax. Mean ± s.d. of n = 3 independent biological replicates.