Sci Transl Med:侏儒症或可通过新方法治疗

2013-10-21 科学网 科学网

软骨发育不全症是基因突变所导致的一种最常见侏儒症。法国研究人员在动物实验中发现,如果注射一种“诱饵蛋白”,让致病基因失去作用,或能治疗软骨发育不全症。 法国卫生和医学研究所的研究人员近日在美国《科学—转化医学》杂志上报告说,软骨发育不全症患者体内的FGFR3基因发生突变后,会导致FGFR3蛋白过度活跃,从而抑制骨骼的正常发育。为此,他们采取一种欺骗策略,让过度活跃的FGFR3蛋白“冷静”下来。

软骨发育不全症是基因突变所导致的一种最常见侏儒症。法国研究人员在动物实验中发现,如果注射一种“诱饵蛋白”,让致病基因失去作用,或能治疗软骨发育不全症。

法国卫生和医学研究所的研究人员近日在美国《科学—转化医学》杂志上报告说,软骨发育不全症患者体内的FGFR3基因发生突变后,会导致FGFR3蛋白过度活跃,从而抑制骨骼的正常发育。为此,他们采取一种欺骗策略,让过度活跃的FGFR3蛋白“冷静”下来。

具体方法是,给软骨发育不全的新生实验鼠多次注射一种叫做sFGFR3的蛋白,这种蛋白可以像“诱饵”一样吸引FGFR3基因的全部“火力”,正常的FGFR3蛋白因此不再受到影响,从而让骨骼发育恢复正常。在他们的实验中,老鼠接受治疗后出现脊椎异常问题的比例从80%降至6%。

研究人员还发现,“诱饵蛋白”疗法还会抑制软骨发育不全实验鼠的瘫痪、呼吸困难等并发症,从而有效降低其死亡率。此外,将这些实验鼠解剖后进行器官、血液等检测,没有发现明显副作用。

尽管还要进行更多研究以确定人类患者利用sFGFR3蛋白治疗的安全性与有效性,但研究人员认为,软骨发育不全症患儿在出生第一年内接受这种治疗,骨骼生长很有可能恢复正常化。

原文检索

Garcia S, Dirat B, Tognacci T, Rochet N, Mouska X, Bonnafous S, Patouraux S, Tran A, Gual P, Le Marchand-Brustel Y, Gennero I, Gouze E.Postnatal Soluble FGFR3 Therapy Rescues Achondroplasia Symptoms and Restores Bone Growth in Mice. 2013 Sep

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