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. 2016 Mar 8;24(5):855–866. doi: 10.1038/mt.2016.33

Figure 1.

Figure 1

Development of a single-stranded adeno-associated virus (AAV) construct containing IGHMBP2 and expression of IGHMBP2 in vivo. (a) 2.9 kb of human IGHMBP2 cDNA under control of the ubiquitously expressing chicken-β-actin (CBA) promoter along with an optimized intron within the 5' leader sequence and a synthetic polyA site were cloned into a single stranded AAV vector. (b,c) Western blot reveals increased levels of IGHMBP2 expression in spinal cord of homozygous nmd mice 30 days post-intracerebroventricular (ICV) injection of 2 × 1011 viral genomic copies. Tissue extracts of “Het” was used as positive control and anti-β-actin was used as loading control. (d,e) Western blot detects elevated IGHMBP2 expression in spinal cord extracts of low- and high-dose-treated SMARD1 mice 10 days post-ICV injection of 1.25 × 1011 (low-dose) and 2.5 × 1011 (high-dose) viral genomic copies. Tissue extract of WT was used as positive control and anti tubulin was used as loading control. HEK293 cells transfected with IGHMBP2 construct was utilized as size control in both gels. The expression level was normalized to IGHMBP2 levels in “Het” (c) and “WT” (e).