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Assay and Drug Development Technologies|August 23, 2014
Assays for the identification and prioritization of drug candidates for spinal muscular atrophyJonathan J Cherry, Dione T Kobayashi, Maureen M Lynes, et al.Epilepsia|May 13, 2023
Functional evaluation of epilepsy-associated KCNT1 variants in multiple cellular systems reveals a predominant gain of function impact on channel propertiesChristopher A Hinckley, Zhonghua Zhu, Jen-Hwa Chu, et al.EMBO Molecular Medicine|June 7, 2013
Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compoundsJonathan J Cherry, Erkan Y Osman, Matthew C Evans, et al.Nucleic Acids Research|October 31, 2021
Targeting 3' and 5' untranslated regions with antisense oligonucleotides to stabilize frataxin mRNA and increase protein expressionYanjie Li, Jixue Li, Jun Wang, et al.JCI Insight|November 25, 2016
ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in miceMahlet B Abera, Jingbo Xiao, Jonathan Nofziger, et al.Bioorganic & Medicinal Chemistry Letters|November 7, 2017
Optimization of a series of heterocycles as survival motor neuron gene transcription enhancersSungwoon Choi, Alyssa N Calder, Eliza H Miller, et al.Journal of Medicinal Chemistry|May 9, 2017
Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular AtrophyAnne Rietz, Hongxia Li, Kevin M Quist, et al.Journal of Medicinal Chemistry|March 4, 2017
Design of Potent mRNA Decapping Scavenger Enzyme (DcpS) Inhibitors with Improved Physicochemical Properties To Investigate the Mechanism of Therapeutic Benefit in Spinal Muscular Atrophy (SMA)Ariamala Gopalsamy, Arjun Narayanan, Shenping Liu, et al.Plos One|September 26, 2017
In vitro and in vivo effects of 2,4 diaminoquinazoline inhibitors of the decapping scavenger enzyme DcpS: Context-specific modulation of SMN transcript levelsJonathan J Cherry, Christine J DiDonato, Elliot J Androphy, et al.Pageof 2