Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy
J. Clin. Invest. 117:12 doi:10.1172/JCI33355 [Go to this article.]

Figure 5
Antisense morpholino represses exon 7a inclusion, restores ClC-1 protein expression, and rescues myotonia in Mbnl1^ΔE3/ΔE3 mice.

(A) RT-PCR shows reduced inclusion of exon 7a at 3 weeks after injection of antisense morpholino 1 (20 μg antisense or invert control). (B) Quantitation of splicing results shown in A as mean ± SD; n = 3 per group; **P < 0.001, antisense versus invert-treated control; t test. Immmunofluorescence for ClC-1 is increased 3 weeks after injection with antisense (D) as compared with invert-treated control (C). Scale bar: 20 μm. (E) Myotonia in Mbnl1^ΔE3/ΔE3 TA muscle was reduced 3 weeks after treatment with antisense morpholino but not in muscle treated with invert control. Mean ± SD; n = 3 per group; **P < 0.0001, antisense versus invert-treated control; ANOVA.