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Molecular Therapy : the Journal of the American Society of Gene Therapy|February 11, 2016
Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose DependentMonir Shababi, Zhihua Feng, Eric Villalon, et al.The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|April 17, 2015
Non-aggregating tau phosphorylation by cyclin-dependent kinase 5 contributes to motor neuron degeneration in spinal muscular atrophyNimrod Miller, Zhihua Feng, Brittany M Edens, et al.Human Molecular Genetics|August 9, 2021
SMN protein is required throughout life to prevent spinal muscular atrophy disease progressionXin Zhao, Zhihua Feng, Nicole Risher, et al.Human Molecular Genetics|February 8, 2013
Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular AtrophyMelissa S Cobb, Ferril F Rose, Hansjörg Rindt, et al.Human Molecular Genetics|June 4, 2013
The DcpS inhibitor RG3039 improves motor function in SMA miceJames P Van Meerbeke, Rebecca M Gibbs, Heather L Plasterer, et al.Neuron|March 2, 2023
A spinal muscular atrophy modifier implicates the SMN protein in SNARE complex assembly at neuromuscular synapsesJeong-Ki Kim, Narendra N Jha, Tomoyuki Awano, et al.American Journal of Human Genetics|March 26, 2019
ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial DysautonomiaElisabetta Morini, Dadi Gao, Connor M Montgomery, et al.Human Molecular Genetics|March 3, 2016
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophyXin Zhao, Zhihua Feng, Karen K Y Ling, et al.Human Molecular Genetics|July 18, 2012
Characterization of behavioral and neuromuscular junction phenotypes in a novel allelic series of SMA mouse modelsMelissa Osborne, Daniel Gomez, Zhihua Feng, et al.Journal of Medicinal Chemistry|July 26, 2018
Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)Hasane Ratni, Martin Ebeling, John Baird, et al.Pageof 5