The present invention relates to antisense oligonucleotides or oligonucleotide analogues adapted to target pre-mRNA which encodes GNB3s and prevent aberrant splicing events. In particular the invention relates to non-natural oligonucleotides, especially morpholino oligonucleotides. Such oligonucleotides have the potential to treat various medical conditions. The invention also provides, pharmaceutical compositions, methods of using the oligonucleotides, for example in medical treatments.
|IPC||C12N 15/00; A61K 31/00; A61P 43/00|
|Publication status||Published - 20 Nov 2013|