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Methods and means for treating genetic disorders associated with instability of DNA repeats
Methods and means for treating genetic disorders associated with instability of DNA repeats
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机译:用于治疗与DNA重复序列不稳定相关的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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