Fig. 3: In vivo prime editing using pegRNA-AAV and PE mRNA–LNP.
From: Treatment of a metabolic liver disease in mice with a transient prime editing approach
a, Schematic illustration of the experimental set-up. b, Editing rates (left) and indel rates (right) at the Dnmt1 locus in untreated tail tissue or mice treated with a single dose of 3 mg kg−1 PE2 or PEmax mRNA–LNP (n = 3). c, Pahenu2 correction rates (left) and indel rates (right) of untreated tail tissue (n = 5) or mice treated with a single dose of 3 mg kg−1 PE2 or PEmax mRNA–LNP (n = 4). In b and c, values represent mean ± s.d. of independent biological replicates and were analysed for the indels using unpaired two-sided Student’s t-tests (P > 0.05) d, Editing rates (blue line, left y axis) and indel rates (grey bar, right y axis) of mice treated with one dose of 3 mg kg−1 PEmax mRNA–LNP and pretreatment of different doses of scAAV encoding the Dnmt1-targeting pegRNA (n = 3). Values represent mean ± s.e.m. e, Editing rates (top) and indel rates (bottom) of mice pretreated with scAAV encoding the Dnmt1-targeting pegRNA and with one dose of 2 mg kg−1 PEmax mRNA–LNP and active Vpx or inactive Vpx (dVpx) coding mRNA (n = 3). In b, c and e, values represent mean ± s.d. of independent biological replicates.
