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Single-stranded oligonucleotides, which are complementary to reactive elements, for the treatment of DNA reuptake instability-associated diseases
Single-stranded oligonucleotides, which are complementary to reactive elements, for the treatment of DNA reuptake instability-associated diseases
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机译:与反应性元件互补的单链寡核苷酸,用于治疗与DNA再摄取不稳定性相关的疾病
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摘要
The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to modified oligonucleotides which can be applied in method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.
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