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靶向 EZH2:杜氏肌营养不良症治疗新曙光
目前,虽然基因编辑疗法为 DMD 的治疗带来了一丝希望,但临床应用的道路上仍布满荆棘,比如难以确定临床疗效、无法有效控制潜在的脱靶效应等。而糖皮质激素(如地夫可特、泼尼松)虽能延缓疾病进展,却也伴随着肥胖、行为改变、身材矮小和骨质减少等诸多副作用,就像 “饮鸩止渴”,让患者陷入两难境地。因此,寻找更安全有效的治疗方法,成为了医学领域亟待攻克的难题。
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