New isolated antisense-oligonucleotide comprising sequence that is hybridized to messenger RNA-splicing-sequence of mutation-bearing exons of pre-messenger RNA of titin-gene and induces skipping of exons, used to treat heart disease

摘要

Isolated antisense-oligonucleotide (AO) comprising a sequence that is specifically hybridizable to a messenger RNA (mRNA)-splicing-sequence of mutation-bearing exons of pre-mRNA of titin-gene and is able to induce a skipping of the exons is new. ACTIVITY : Cardiant; Cardiovascular-Gen. MECHANISM OF ACTION : Angiotensin-converting enzyme inhibitor; Beta-blocker.