Drug screening with human SMN2 reporter identifies SMN protein stabilizers to correct SMA pathology.-论文-万方医学网

第一作者： Yiran,Wang

第一单位： Brain and Spinal Cord Innovative Research Center, Tongji Hospital, Tongji University School of Medicine, Shanghai, China.

作者： Yiran,Wang ^[1] ; Chongchong,Xu ^[2] ; Lin,Ma ^[3] ; Yongchao,Mou ^[2] ; Bowen,Zhang ^[1] ; Shanshan,Zhou ^[1] ; Yue,Tian ^[1] ; Jessica,Trinh ^[4] ; Xiaoqing,Zhang ^[5] ; Xue-Jun,Li ^[6]

作者单位： Brain and Spinal Cord Innovative Research Center, Tongji Hospital, Tongji University School of Medicine, Shanghai, China. ^[1] Department of Biomedical Sciences, University of Illinois College of Medicine Rockford, Rockford, IL, USA.;Department of Bioengineering, University of Illinois at Chicago, Chicago, IL, USA. ^[2] Brain and Spinal Cord Innovative Research Center, Tongji Hospital, Tongji University School of Medicine, Shanghai, China.;Key Laboratory of Reconstruction and Regeneration of Spine and Spinal Cord Injury, Ministry of Education, Shanghai, China.;Key Laboratory of Neuroregeneration of Shanghai Universities, Tongji University, School of Medicine, Shanghai, China. ^[3] Department of Biomedical Sciences, University of Illinois College of Medicine Rockford, Rockford, IL, USA. ^[4] Brain and Spinal Cord Innovative Research Center, Tongji Hospital, Tongji University School of Medicine, Shanghai, China xqzhang@tongji.edu.cn.;Key Laboratory of Reconstruction and Regeneration of Spine and Spinal Cord Injury, Ministry of Education, Shanghai, China.;Key Laboratory of Neuroregeneration of Shanghai Universities, Tongji University, School of Medicine, Shanghai, China.;Tsingtao Advanced Research Institute, Tongji University, Shanghai, China.;Shanghai Institute of Stem Cell Research and Clinical Translation, Shanghai, China.;Translational Medical Center for Stem Cell Therapy, Shanghai East Hospital, Tongji University School of Medicine, Shanghai, China. ^[5] Department of Biomedical Sciences, University of Illinois College of Medicine Rockford, Rockford, IL, USA xjli23@uic.edu.;Department of Bioengineering, University of Illinois at Chicago, Chicago, IL, USA. ^[6]

医学主题词动物(Animals);血脑屏障(Blood-Brain Barrier);半胱氨酸蛋白酶抑制剂(Cysteine Proteinase Inhibitors);二肽类(Dipeptides);疾病模型, 动物(Disease Models, Animal);药物评价, 临床前(Drug Evaluation, Preclinical);基因, 报告(Genes, Reporter);HEK293细胞(HEK293 Cells);人类(Humans);酮类(Ketones);亮氨酸(Leucine);小鼠(Mice);线粒体(Mitochondria);运动神经元(Motor Neurons);肌萎缩, 脊髓性(Muscular Atrophy, Spinal);预防和防护用药(Protective Agents);蛋白质稳定性(Protein Stability);信号传导(Signal Transduction);运动神经元生存蛋白质2(Survival of Motor Neuron 2 Protein);转染(Transfection);治疗结果(Treatment Outcome)

DOI 10.26508/lsa.201800268

PMID 30910806

发布时间 2021-05-14

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Drug screening with human SMN2 reporter identifies SMN protein stabilizers to correct SMA pathology.

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Drug screening with human SMN2 reporter identifies SMN protein stabilizers to correct SMA pathology.

Life science alliance

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Life science alliance

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