Table 2. Editing efficiency and allele specificity of allele-specific CRISPR-Cas9.

Patient-derived iPSC lines were treated with the allele-specific NMD-CRISPR strategy. Subsequently, DNA samples were subjected to PCR to amplify the region harboring rs121918312, and then MiSeq sequencing analysis was performed. The identity of alleles was inferred based on the alleles of rs121918312.

	Allele	Modification	Subject #3 (%)	Subject #6 (%)
NMD-CRISPR using PAS	Mutant	Modified	4.6	4.1
		Unmodified	41.8	43.4
	Normal	Modified	0	0
		Unmodified	53.6	52.5