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Table 1 Comparison of the mutation correction efficacies, off-target, and indels rates between CRISPR/Cas9-HDR and ABEs

From: In vitro genome editing rescues parkinsonism phenotypes in induced pluripotent stem cells-derived dopaminergic neurons carrying LRRK2 p.G2019S mutation

 

HDR

ABE

Number of colonies

47

53

Insertions or deletions on exon 41

27 (57.4%), all are small deletions

0

Creating additional missense mutations

0

14 (26.4%):

11 (20.75%) are within active window

3 (5.67%) are proximal off-target editing

Genotype: still LRRK2 c.6055G > A mutation without correction

17 (36.2%)

26 (49.1%)

Genotype: corrected (LRRK2 c.6055A > G)

3 (6.4%)

13 (24.5%)

  1. HDR, Homology-directed repair; ABE, adenine base editing