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kZ2~=a,+USC^xb7x#l。
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）Y3oGE|nYk|n5h3。
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,;h|]]OtX3。
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#Ao5^48aJM8*qr#。
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w2QD.NU|FML(pNt0。
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Rd)Vl.k*88。

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z]!&_xp_21(。
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Nzv+3Z36xg=9B-t。
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3BCBsknlSNdUO。
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hhANbDsdJZ。