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Methods and means for treating DNA repeat instability associated genetic disorders
Methods and means for treating DNA repeat instability associated genetic disorders
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机译:治疗DNA重复不稳定性相关遗传疾病的方法和手段
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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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