Fig. 1

Premature death, absence of dystrophin expression and muscle atrophy in R-DMDdel52 rats. a Scheme of CRISPR-mediated gene deletion in rats and genotyping data from WT and R-DMDdel52 animals. Position of WT and deletion (del52) bands is indicated. b Pictures of R-DMDdel52 rats (left) and a WT littermate (right) aged 12 months, showing a marked facial muscle atrophy that modifies the appearance of the animal. c Kaplan Meier Curve for the frequency of WT (black curve) and R-DMDdel52 (red curve) rat survival. d Representative immunofluorescence for DYSTROPHIN (orange) and ßDYSTROGLYCAN (red) in tibialis anterior sections of 3-month-old (left panels) and 12-month-old (right panels) WT and R-DMDdel52 rats. Scale bar 20 μm. e Quantification of the percentage of dystrophin-positive fibres in WT and R-DMDdel52 TA at 3 weeks, 3, 6, 12 months of age. f Western blot analysis of WT and R-DMDdel52 proteins extracted from soleus and diaphragm tissues. The amount of loaded proteins was normalized on β-TUBULIN levels. g CK levels of WT and R-DMDdel52 rats aged 3 weeks, 3 months, 6 months and 12 months. h Weight curve showing that R-DMDdel52 rats exhibited a progressive loss of weight compared to their healthy littermates (WT). i) Graph showing the body and tibial length in centimetres of R-DMDdel52 and WT rats. j) Pictures of R-DMDdel52 muscles at 12 months of age: tibialis anterior, extensor digitorum longus, soleus, and heart. k) Graph showing the weight in grams of different skeletal muscles from R-DMDdel52 rats and WT aged 12 months