Extended Data Fig. 2: Early intervention to upregulate Lama1 prevents disease progression in dy2j/dy2j mice.
From: A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene
a, P2 neonatal dy2j/dy2j mice were injected with AAV9s carrying no guide (n = 6; 7.5 × 1011 viral genomes) or three guides (n = 4; split into two vectors; total dose of 2 × 7.5 × 1011 viral genomes) in the temporal vein and euthanized 7 weeks later. b, c, Immunofluorescence staining shows Lama1 expression in tibialis anterior (b) and gastrocnemius (c) muscles. Scale bars, 100 µm. d, e, General muscle histopathology was evaluated by haematoxylin and eosin staining on tibialis anterior (d) and gastrocnemius (e) muscles. Scale bars, 200 µm. All mice (n = 6, no guide and n = 4, three guides) were analysed for experiments shown in b–e, which were repeated three times independently with similar results. f–i, Fibrosis (f, h) and minimum Feret diameter (g, i) were quantified for all mice from both treatment groups (n = 6, no guide and n = 4, three guides) by two independent experimenters with similar results. Data are mean ± s.d. **P = 0.0025 (f), *P = 0.0103 (g), *P = 0.0142 (h), P = 0.0711 (i); two-tailed Student’s t-test.
