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剪接开关反义寡核苷酸诱导的 SMA 患者成纤维细胞外显子夹杂的评价
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剪接开关反义寡核苷酸诱导的 SMA 患者成纤维细胞外显子夹杂的评价

Article DOI: 10.3791/57530-v • 07:02 min • May 11th, 2018
May 11th, 2018 •
1, 1, 1,2
1Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, 2Muscular Dystrophy Canada Research Chair, Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry

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各种反义寡核苷酸 (AONs) 被证明可以诱导外显子夹杂 (剪接调制) 和抢救 SMN 表达的脊髓肌萎缩 (SMA)。在这里, 我们描述了一个协议, 以 lipotransfection 诱导外显子包含在SMN2基因和评价方法, 以确定的有效性, SMA 患者成纤维细胞。

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药物 问题 135 脊髓肌萎缩 (sma) 反义寡核苷酸 锁定核酸 (低噪声) 反义治疗 运动神经元的生存 (SMN) 外显子夹杂 剪接调制 nusinersen Werdnig 霍夫曼病 (sma 1) Dubowitz疾病 (sma 2) Kugelberg-Welander 病 (sma 3) 剪接交换寡核苷酸 (SSO)
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