Table 1.

Gene-Targeting Frequencies

HDAd	No. of Cells Transduced	No. of puroR Colonies	Freq puroR	Correct Gene Targeting	Gene Correction	One Allele Targeted	Both Alleles Targeted	Indels in Non-targeted Allele
HD-23.8-CFTRm-PACTk-DTA	2 × 106	30	1.5 × 10−5	NDa	NDb	NDc	NDd	NA
HD-23.8-CFTRm-PACTk-DTA+Cas9−28	2 × 106	1,937	9.7 × 10−4	100% (21/21)	93.8% (15/16)	100% (21/21)	0% (0/21)	75% (15/20)
HD-23.8-CFTRm-PACTk-DTA+Cas9+0	2 × 106	3,501	1.8 × 10−3	95.7% (22/23)	60% (12/20)	90.9% (20/22)	9.1% (2/22)	100% (21/21)

Freq, Frequency; NA, not applicable; ND, not determined.

^aND in this study but was 89.5% in a prior study with the same HDAd.¹⁴

^bND in this study but was 100% in a prior study with the same HDAd.¹⁴

^cND in this study but was 100% in a prior study with the same HDAd.¹⁴

^dND in this study but was 0% in a prior study with the same HDAd.¹⁴