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![[Figure 5]](cb5061fig5mag.jpg)
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Figure 5
BuDNs targeting the HBB gene are accurate and highly active. (a) Two BuD arrays targeting a DNA region within the HBB gene near a locus known to be responsible for sickle-cell anaemia were generated. The arrays were fused to a FokI domain and transfected into HEK293 human cells (see Methods ![[link]](../../../../../../logos/arrows/d_arr.gif) ). (b) The efficiency of the double-strand breaks induced by the BuDNs was monitored using the T7 endonuclease assay. (c) The genomic DNA was also analyzed by deep sequencing. The most representative indels identified are reported in the table. Insertions are depicted in red and deletions by dashes. (d) Table quantifying the targeted mutagenesis events at the HBB locus by deep sequencing (see Supporting Information). (e) Targeted gene insertion (TGI) frequency determined at the HBB locus in the presence of the donor DNA transfected with or without BuDNs. See Supplementary Fig. S11 and Supporting Information for a detailed description. |
![[Figure 5]](cb5061fig5.jpg)
 | STRUCTURAL BIOLOGY |
ISSN: 2059-7983
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