Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G <sub>4</sub> C <sub>2</sub> ) repeat expansion in vitro and in vivo ALS models
Jessica A. Bush, Haruo Aikawa, Rita Fuerst, Yue Li, Andrei Ursu, Samantha M. Meyer, Raphael I. Benhamou, Jonathan L. Chen, Tanya Khan, Sarah Wagner-Griffin, Montina J. Van Meter, Yuquan Tong, Hailey Olafson, Kendra McKee, Jessica L. Childs‐Disney, Tania F. Gendron, Yong‐Jie Zhang, Alyssa N. Coyne, Eric T. Wang, Ilyas Yildirim, Kye Won Wang, Leonard Petrucelli, Jeffrey D. Rothstein, Matthew D. Disney
Abstract
is a strategy for mitigating c9ALS/FTD-associated pathologies and studying disease-associated pathways in preclinical models.
Topics & Concepts
In vivoNucleaseIn vitroRNATrinucleotide repeat expansionSmall moleculeRibonucleaseEndogenyBiologyChemistryMolecular biologyCell biologyGeneticsComputational biologyBiochemistryGeneAlleleNeurogenetic and Muscular Disorders ResearchAmyotrophic Lateral Sclerosis ResearchGenetics and Neurodevelopmental Disorders