Table 1

Molecular outcomes from treatment of HSPCs with ZFNs and an oligonucleotide donor

OutcomeFrequency (%)
No change 46.1 
NHEJ-mediated deletions 30.5 
Oligonucleotide-templated gene modification 18.8 
NHEJ-mediated insertions 2.8 
Half gene modification, half NHEJ 1.5 
Capture of the donor oligonucleotide 0.4 
OutcomeFrequency (%)
No change 46.1 
NHEJ-mediated deletions 30.5 
Oligonucleotide-templated gene modification 18.8 
NHEJ-mediated insertions 2.8 
Half gene modification, half NHEJ 1.5 
Capture of the donor oligonucleotide 0.4 
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