Echocardiographic measurements of LVPW thickness, LVPW/LVDd ratio and FS (defined in Fig. 2 ) in R403Q-129SvEv/S4 mice at 20 weeks of age treated with low (1.1 x 10 12 vg kg -1 , n = 1 male, 2 females), medium (5.4 x 10 12 vg kg -1 , n = 3 females) and high (2.2 x 10 13 vg kg -1 , n = 6 females) doses of AAV9-Cas9, compared to untreated WT 129SvEv/S4 ( n = 3 females) and untreated R403Q-129SvEv/S4 ( n = 4 females) mice. Note that one mouse treated with the low dose had increased LVPW and increased LVPW/LVDd, indicating a non-therapeutic response. Data are presented as mean values s.d., and significance was assessed by two-tailed t -test ( Supplementary Information ).
