Antisense‐mediated exon skipping: a therapeutic strategy for titin‐based dilated cardiomyopathy. Issue 5 (10th March 2015) - Gramlich, Michael
Abstract Truncating mutations in the giant sarcomeric protein titin (TTN) are a major cause for inherited forms of dilated cardiomyopathy (DCM). Antisense oligonucleotides (AON)‐mediated exon skipping is shown here to be a promising strategy as potential treatment option for DCM.