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Splice-Switching Antisense Oligonucleotides Reduce LRRK2 Kinase Activity in Human LRRK2 Transgenic Mice

Parkinson’s disease (PD) is a progressive neurological disorder estimated to affect 7–10 million people worldwide. There is no treatment available that cures or slows the progression of PD. Elevated leucine-rich repeat kinase 2 (LRRK2) activity has been associated with genetic and sporadic forms of...

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Foilsithe in:Mol Ther Nucleic Acids
Main Authors: Korecka, Joanna A., Thomas, Ria, Hinrich, Anthony J., Moskites, Alyssa M., Macbain, Zach K., Hallett, Penelope J., Isacson, Ole, Hastings, Michelle L.
Formáid: Artigo
Teanga:Inglês
Foilsithe: American Society of Gene & Cell Therapy 2020
Ábhair:
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC7393423/
https://ncbi.nlm.nih.gov/pubmed/32736291
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2020.06.027
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