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

• Published in: Molecular therapy. Nucleic acids Vol. 21; pp. 623 - 635
• Main Authors: Korecka, Joanna A., Thomas, Ria, Hinrich, Anthony J., Moskites, Alyssa M., Macbain, Zach K., Hallett, Penelope J., Isacson, Ole, Hastings, Michelle L.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 04.09.2020; American Society of Gene & Cell Therapy; Elsevier
• Subjects: antisense oligonucleotide; LRRK2; LRRK2 BAC mice; Parkinson’s disease; RAB10; splicing; RAB10; splicing; antisense oligonucleotide; LRRK2 BAC mice; LRRK2; Parkinson’s disease

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...