摘要 : Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by homozygous deletions or mutations in survival motor neuron gene 1 (SMN1). Currently, the primary therapeutic strategy for SMA is to increase t... 展开
作者 | Wang~ Jia Bai~ Jinli OuYang~ Shijia Wang~ Hong Jin~ Yuwei Peng~ Xiaoyin Ge~ Xiushan Jiao~ Hui Zou~ Jizhen He~ Cai Xiao~ Ping Song~ Fang Qu~ Yujin |
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作者单位 | |
期刊名称 | 《Human Molecular Genetics》 |
总页数 | 16 |
语种/中图分类号 | 英语 / R394 |
关键词 | HISTONE DEACETYLASE DNA METHYLATION DISEASE SEVERITY SURVIVAL PROTEIN NUSINERSEN DISCOVERY CHILDREN THERAPY PHASE-1 |
馆藏号 | N2007EPST0000828 |