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神经肌肉疾病治疗新进展:寡核苷酸与偶联疗法展现潜力
寡核苷酸疗法作为一种靶向RNA的创新治疗模式,在治疗包括杜氏肌营养不良症(DMD)在内的多种神经肌肉疾病中已经展现出显著疗效。本文将重点介绍在MDA临床与科学大会上的部分寡核苷酸疗法的进展。
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