Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
AssigneesRosalind Franklin University of Medicine and Science, The McLean Hospital Corporation
InventorsMichelle L. Hastings, Ole Isacson, Joanna A. Korecka-Roet
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Abstract
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.
Claims16
A method of modulating splicing or expression of a LRRK2 transcript in a cell comprising contacting the cell with at least one compound comprising a modified oligonucleotide having 8 to 30 linked nucleosides and having a nucleobase sequence comprising a complementary region, wherein the complementary region comprises at least 8 contiguous nucleobases complementary to an equal-length portion of a target region of a Leucine-Rich-Repeat-Kinase (LRRK2) transcript, wherein the modified oligonucleotide has a nucleobase sequence as set forth in SEQ ID NO:1, SEQ ID NO:2, SEQ ID NO:6, SEQ ID NO:7, or SEQ ID NO:8.
Dependencies are inferred from the claim text.