Table 2.

On-target efficiency compared to off-target mutation rate.

Gene	Reagents	Source of gDNA	On-Target Modification Frequency	Off-Target Modification Frequency	Reference
CCR5	tcPNA/DNA	THP-1 cells	2.8%	<0.057%	[17]
CCR5	tcPNA/DNA PLGA NPs	human PBMCs	0.97%	0.004%	[19]
CCR5	tcPNA/DNA PLGA NPs	humanized NOD-scid IL2rγnull mouse bone marrow	0.43%	0.004%	[18]
CFTR	tcPNA/DNA PBAE/PLGA/MPG NPs	CFBE cells	9.2%	<0.00001%	[21]
CFTR	tcPNA/DNA PBAE/PLGA/MPG NPs	mouse nasal epithelium	5.7%	<0.0001%	[21]
β-globin	γtcPNA/DNA PLGA NPs with SCF	total mouse bone marrow cells	3.9%	0.0032%	[22]
β-globin	γtcPNA/DNA PLGA NPs with SCF	human CD34+ cells	5.02%	0.000012%	[22]