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Treatment of Down syndrome gene-related diseases by repression of natural antisense transcripts for Down syndrome genes

机译:通过抑制唐氏综合症基因的天然反义转录本治疗唐氏综合症基因相关疾病

摘要

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Down Syndrome Gene, in particular, by targeting natural antisense polynucleotides of a Down Syndrome Gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Down Syndrome Genes.
机译:本发明涉及反义寡核苷酸,其特别是通过靶向唐氏综合症基因的天然反义多核苷酸来调节唐氏综合症基因的表达和/或功能。本发明还涉及这些反义寡核苷酸的鉴定及其在治疗与唐氏综合症基因表达有关的疾病和病症中的用途。

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