公开(公告)号：EP2707486A4

公开(公告)日：2015-02-18

申请号：EP12767650

申请日：2012-04-04

申请人： ZAIN LUQMAN RULA

发明人： BERGQUIST HELEN ,  GOOD LIAM

IPC分类号： C12Q1/68 ,  A61K31/712 ,  C12N15/113

CPC分类号： C12N15/113 ,  A61K31/712 ,  C12N2310/11 ,  C12N2310/3181 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2320/30 ,  C12Q1/6883 ,  C12Q2600/156

摘要： The present invention is directed to oligonucleotides based on peptide nucleic acid oligonucleotide or an equivalent oligonucleotide analogue, such as morpholino or a locked nucleic acid sequences and the use of such oligonucleotides for the dissociation of higher order structures, including triplex-helix DNA structures, in repeated sequences of DNA in Friedreich's ataxia. The dissociation of such structures may be used in the diagnosis and/or treatment of Friedreich's ataxia. Consequently, the present invention is also directed to a method for diagnosing Friedreich's ataxia and the use of peptide nucleic acid oligonucleotide or an equivalent oligonucleotide analogue, such as morpholino or a locked nucleic acid sequences in the treatment of Friedreich's ataxia. Preferably, the oligonucleotides comprise a sequence selected from the group consisting of (GAA)n, (CTT)n, (JTT)n or a mixed (JTT/CTT)n sequence.