Table 1 Gaac.1935 guide RNA on-target activity and HDR efficiency.

From: CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease

  1. The target sequence, PAM motifs and donor templates used for testing of Gaac.1935 guide RNAs in C2C12 mouse mybloasts are outlined. Gaac.1935 locus for each gRNA target sequence is highlighted in yellow. Desired Gaac.1935 KI mutation (red), silent PAM site (either green or gold, corresponding to gRNA) and gRNA seed region (black) mutations are bolded and underlined in the donor template sequence. Total on-target Cas9 nuclease activity and HDR efficiency for each Gaac.1935 guide RNA condition is displayed as the average of two independent experiments.
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