DIAGNOSIS AND TREATMENT OF FRIEDREICH'S ATAXIA
    32.
    发明公开
    DIAGNOSIS AND TREATMENT OF FRIEDREICH'S ATAXIA 审中-公开
    诊断和治疗弗里德共济失调

    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

    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.