Extended Data Fig. 2: Reduction of blood phenylalanine levels by PAH1 hybrid guide RNAs.
From: Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs
Blood phenylalanine levels in homozygous PAH P281L mice (n = 4–5 animals per group; refer to Source Data file for exact numbers) before and 48 hours after treatment with 2.5 mg/kg dose of LNPs with PAH1 standard or hybrid gRNA in combination with ABE8.8 mRNA (1 blood sample per timepoint). The dotted line marks the upper end of the normal range for blood phenylalanine, 125 µmol/L. Means are shown; P values were calculated with the two-sided Mann–Whitney U test.