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Spinal muscular atrophy, or SMA, is an inherited disease that affects a child's ability to move muscles.
脊髓性肌萎缩症，简称SMA，是一种影响儿童肌肉运动能力的遗传性疾病。
A gene involved in the development of SMA is called SMN1.
与SMA病情相关的基因是被称为“运动神经元存活1号”（SMN1）。
SMN1 produces survival motor neuron protein that helps motor neurons to develop connections between the brain and the muscles.
SMN1基因的作用是产生“运动神经元存活”蛋白质，该蛋白质是帮助运动神经元发育大脑和肌肉之间的连接。
Babies born with two mutated copies of SMN1 gene cannot develop proper brain-muscle connections.
生下来两个SMN1副本就发生基因突变的婴儿，是无法发育正常的大脑-肌肉连接的。
They lose their ability to move, talk and eventually breathe.
他们会丧失移动、说话，最终是呼吸的能力。
Zolgensma, a gene therapy developed by AveXis, is a therapy that fixes SMA at the genetic level.
由AveXis公司研发的Zolgensma，是一种在遗传层面上治疗SMA的基因疗法。
Zolgensma is an adeno-associated virus that carries a functional copy of SMN1 gene.
Zolgensma通过腺相关病毒载体递送SMN1基因副本。
Following intravenous administration, Zolgensma viral particles travel to the brain, cross blood-brain barrier and infect the brain cells.
静脉注射后，Zolgensma病毒颗粒会进入大脑，穿过血脑屏障，将基因传递给脑细胞。
DNA with SMN1 gene delivered by the associated virus, settles in cell nucleus and starts producing SMN protein.
以病毒为载体、含有SMN1基因的DNA，会在细胞核内定居并开始产生SMN蛋白。
Babies treated with Zolgensma start gaining motor strengths and reaching their developmental milestones,
接受Zolgensma治疗的婴儿开始获得运动力量，达到发育运动里程碑，
they get a chance to live healthy lives that we all take for granted.
从而有机会过上我们认为理所当然的健康生活。
