Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
摘要
<p>Provided herein are peptide-linked morpholino (PPMO) antisense oligonucleotides that target the poly CUG repeat tract in the 3' untranslated region of the gene encoding dystrophia myotonica-protein kinase (DMPK) and methods for systemic administration of the same for the treatment of mytonic dystrophy type I (DM1).</p>
申请公布号
AU2013323820(A1)
申请公布日期
2015.04.16
申请号
AU20130323820
申请日期
2013.09.24
申请人
GENZYME CORPORATION
发明人
LEGER, ANDREW;WENTWORTH, BRUCE;NELSON, CAROL A.;WEEDEN, TIMOTHY E.;CLAYTON, NICHOLAS;CHENG, SENG