Fig. 2: In vivo treatment efficacy of AAV8-HLP-mGHR in Laron mice.
From: First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model
a Timeline summarized the experiment performed. b Representative photos for uninjected control mice (GHR+/+, GHR+/− and GHR−/−) and Laron mice (GHR−/−) injected with 4 × 1010 vg/mouse of AAV8-HLP-Luc and AAV8-HLP-mGHR in male (i) and female (ii). Original unmodified image was shown in Supplementary Fig. S1. Weekly measurement of (c) body weight and (d) body length post AAV injection and the corresponding (e) normalized body weight and (f) normalized body length. Body weight and length were normalized to 100% at the time of AAV injection to correct the initial body weights and length variation of mice. AAV8-HLP-mGHR and AAV8-HLP-Luc treated groups were monitored for 25–26 weeks post AAV injection while uninjected control groups were monitored for 16 weeks only. Data were presented as mean ± s.e.m. n = 5–6. Statistical significances were determined by two-way ANOVA where **** indicates p < 0.0001 and considered extremely significant.
