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
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Figure 1
Design of antisense morpholinos.

(A) Inclusion of ClC-1 exon 7a induces a frame shift and premature termination codon in exon 7. Annealing of antisense morpholino to the 3′ splice site of exon 7a in the ClC-1 pre-mRNA is intended to prevent spliceosomal recognition of this exon. (B) Alignment of ClC-1 pre-mRNA (top strand) with antisense morpholinos targeting the 3′ or 5′ splice sites of exon 7a is shown. The control morpholino is the 5′-3′ invert of the 3′ splice site blocker. Exonic sequences are in upper case, intronic sequences are in lower case.