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Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease

机译:针对富含亮氨酸的重复激酶2(LRRK2)的反义化合物,用于治疗帕金森病

摘要

The present disclosure relates generally to compounds comprising oligonucleotides complementary to a Leucine-Rich-Repeat-Kinase (LRRK2) RNA transcript. Certain such compounds are useful for hybridizing to a LRRK2 RNA transcript, including but not limited to a LRRK2 RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the LRRK2 transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Parkinson's disease.
机译:本公开总体上涉及包含与亮氨酸-富集-重复激酶(LRRK2)RNA转录物互补的寡核苷酸的化合物。某些此类化合物可用于与LRRK2 RNA转录本杂交,包括但不限于细胞中的LRRK2 RNA转录本。在某些实施方案中,这样的杂交导致LRRK2转录物的剪接的调节。在某些实施方案中,此类化合物用于治疗与帕金森氏病有关的一种或多种症状。

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