Extended Data Fig. 3: Upregulation of Lama1 corresponds to improvement of muscle functions.
From: A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene
Three-week-old dy2j/dy2j mice were injected systemically in the tail vein with AAV9s carrying no guide (n = 9) at the dose of 3 × 1011 viral genomes (vg) per gram of mouse, or three guides at different doses of 7.5 × 1010 (n = 4), 1.5 × 1011 (n = 3) or 3 × 1011 (n = 8) viral genome per gram of mouse. Two AAVs were needed for the three-guide cohorts, therefore the total doses of virus injected were 1.5 × 1011, 3 × 1011 and 6 × 1011 viral genomes per gram of mouse. a, Tibialis anterior muscles isolated that were isolated four weeks later were stained for Lama1 expression. Asterisks indicate Lama1-positive fibres in the low-dose cohort. The experiment was repeated three times independently with similar results. Scale bars, 100 µm. b, c, In vivo contractile force assays (b) and open field tests (c) were performed at the end of the treatment regimen. Data are mean ± s.d. b, P values from left to right are: P = 0.9849, P = 0.9082, **P = 0.0044; one-way ANOVA with Dunnett’s multiple comparisons test. c, P values from left to right are: P = 0.9640, P = 0.9646, **P = 0.0085; one-way ANOVA with Dunnett’s multiple comparisons test.
