首页>
外国专利>
Diagnosis and treatment of Friedreich's ataxia
Diagnosis and treatment of Friedreich's ataxia
展开▼
机译:弗雷德里希共济失调的诊断和治疗
展开▼
页面导航
摘要
著录项
相似文献
摘要
The present invention is directed to oligonucleotides based on peptide nucleic acid oligonucleotide or an equivalent oligonucleotide analog, 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 analog, 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.
展开▼
机译:本发明涉及基于肽核酸寡核苷酸或等效的寡核苷酸类似物(例如吗啉代或锁定的核酸序列)的寡核苷酸,以及此类寡核苷酸用于解离包括三链螺旋DNA结构在内的更高阶结构的用途。 Friedreich共济失调中重复的DNA序列。这种结构的解离可用于弗里德里希共济失调的诊断和/或治疗。因此,本发明还涉及诊断弗里德里希共济失调的方法,以及在弗里德里希共济失调的治疗中使用肽核酸寡核苷酸或等效的寡核苷酸类似物如吗啉代或锁定的核酸序列。优选地,寡核苷酸包含选自以下的序列:(GAA) n Sub>,(CTT) n Sub>,(JTT) n Sub>或混合(JTT / CTT) n Sub>序列。
展开▼