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Antisense Compounds Targeting Leucine-Rich Repeat Kinase 2(LRRK2) For The Treatment of Parkinsons Disease
Antisense Compounds Targeting Leucine-Rich Repeat Kinase 2(LRRK2) For The Treatment of Parkinsons Disease
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机译:靶向亮氨酸富集重复激酶2(LRRK2)的反义化合物,用于治疗帕金森病
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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.
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