LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate.
第一作者:
Lutz,Garbes
第一单位:
Institute of Human Genetics, University of Cologne, Cologne, Germany.
作者:
主题词
动物(Animals);细胞, 培养的(Cells, Cultured);成纤维细胞(Fibroblasts);基因表达(Gene Expression);人类(Humans);羟肟酸类(Hydroxamic Acids);吲哚类(Indoles);小鼠(Mice);小鼠, 基因敲除(Mice, Knockout);小鼠, 转基因(Mice, Transgenic);肌萎缩, 脊髓性(Muscular Atrophy, Spinal);运动神经元生存蛋白质2(Survival of Motor Neuron 2 Protein);丙戊酸(Valproic Acid)
DOI
10.1093/hmg/ddp313
PMID
19584083
发布时间
2022-02-25
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Human molecular genetics
3645-58页
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