Article
Preclinical and clinical evaluation of the LRRK2 inhibitor DNL201 for Parkinson's disease.
Science translational medicine - 8 Jun 2022
Jennings Danna, Huntwork-Rodriguez Sarah, Henry Anastasia G, Sasaki Jennifer C, Meisner René, Diaz Dolores, Solanoy Hilda, Wang Xiang, Negrou Elvira, Bondar Vitaliy V, Ghosh Rajarshi, Maloney Michael T, Propson Nicholas E, Zhu Yuda, Maciuca Romeo D, Harris Laura, Kay Angela, LeWitt Peter, King T Alex, Kern Drew, Ellenbogen Aaron, Goodman Ira, Siderowf Andrew, Aldred Jason, Omidvar Omid, Masoud Shababa T, Davis Sonnet S, Arguello Annie, Estrada Anthony A, de Vicente Javier, Sweeney Zachary K, Astarita Giuseppe, Borin Marie T, Wong Bradley K, Wong Harvey, Nguyen Hoang, Scearce-Levie Kimberly, Ho Carole, Troyer Matthew D
Abstract excerpt
Mutations in leucine-rich repeat kinase 2 (LRRK2) are the most common genetic risk factors for Parkinson's disease (PD). Increased LRRK2 kinase activity is thought to impair lysosomal function and may contribute to the pathogenesis of PD. Thus, inhibition of LRRK2 is a potential disease-modifying therapeutic strategy for PD. DNL201 is an investigational, first-in-class, CNS-penetrant, selective, ATP-competitive,...
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