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COMPOSITIONS FOR INCREASING SURVIVAL OF MOTOR NEURON PROTEIN (SMN) LEVELS IN TARGET CELLS AND METHODS OF USE THEREOF FOR THE TREATMENT OF SPINAL MUSCULAR ATROPHY
COMPOSITIONS FOR INCREASING SURVIVAL OF MOTOR NEURON PROTEIN (SMN) LEVELS IN TARGET CELLS AND METHODS OF USE THEREOF FOR THE TREATMENT OF SPINAL MUSCULAR ATROPHY
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机译:用于增加靶细胞中运动神经元蛋白(SMN)水平存活的组合物及其治疗脊髓肌萎缩症的方法
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摘要
Compositions for altering splicing activity of the DcpS gene and increasing SMN protein expression in target cells are provided. Also disclosed are methods of use of such compositions for the treatment of SMA.
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