VOA常速英语2019--新研究：镰刀形贫血症或可治愈

Twenty-nine-year-old Lynndrick Holmes of Mobile Alabama says sickle¹ cell anemia² made him feel like his body was suffocating³. He couldn’t do much. So I wanted to play football. I wanted to play basketball. I wanted to do karate⁴ and Taekwondo. But I couldn’t. So you just in this. You just vicariously living through your peers. Most of my life, I just vicariously live through the people that were around me and I watched them live lives that I couldn’t. But no longer. Holmes was one of nine patients in a small clinical trial of a new treatment for sickle cell at the National Institutes of Health. Healthy red blood cells around flat disks that flow easily through the body’s blood vessels⁵. In sickle cell, disease protein crystals formed inside the cells, changing them to a crescent-shape, clogging⁶ vessels and causing pain and organ damage. The disease is caused by a defective⁷ gene⁸.

29岁的林德里克·霍姆斯来自于亚拉巴马州莫比尔县。他说镰刀形红细胞贫血症让他感觉自己的身体要窒息了。他基本什么都做不了。我想踢足球、打篮球、练空手道和跆拳道，但我都做不了。基本就是这样一个状态。这些只能从同龄人的身上间接地感受到。我人生的大部分时间里，我都只能从周围人的身上去感受事情。我会看着他们过一种我无法实践的人生。但以后不会是这样了。霍姆斯和另外8名患者参加了美国国立卫生研究院治疗镰刀形红细胞贫血症的小型临床试验。健康的红细胞是扁平的圆盘状，能够轻易穿过身体各处的血管。而在镰状细胞中，病变蛋白质晶体会在细胞内形成，将细胞改变为新月形，堵塞血管，引发疼痛和器官损伤。这种病是由缺陷的基因引起的。

Rather than editing the gene out, Dr. John Tisdale says his team added a copy of the normal correct gene which produced healthy blood cells. We’re simply adding the gene. We leave the defective gene intact. So this is what’s called gene addition therapy. Within two or three months, all nine patients showed signs of remission, and were starting to discover normal health for the first time in their lives. I’m not the same Lynndrick you know. So I’m like, yo, that happened. I was a part of it and I don’t have sickle cell anymore. NIH director Francis Collins called that spectacular. These patients who had sickle cell disease, now when you look at their blood counts and their blood smears⁹, it looks like they don’t have it anymore. Now it’s early days. We’ve only followed these patients for a year at the most. But it looks like it would be fair to say this could be a cure.

并未将病变基因进行编辑，约翰·蒂斯代尔博士表示，他的团队增加了正常基因的副本，这种正常的基因可以产生健康的血细胞。我们只是增加了基因，没有改变病变基因。这就是所谓的基因添加疗法。不到2-3个月的时间里，这9位病人都有了病情缓解的表现，并首次出现正常的健康状态。我不再是以前的林德里克了，我当时的感受是：哇塞，这一切真地发生了呀。我参加了这次治疗，治好了镰状细胞病。美国国立卫生研究院主任弗朗西斯·柯林斯称之为奇迹。这些镰状细胞病病人，现在如果观测他们的血细胞计数和血涂片，就会发现已经不存在这个问题了。看起来说治愈了是没有问题的。

Not overnight, he adds. And with the technology and cost involved in this trial, it’s unlikely to help sickle cell patients in poor countries. But the approach of introducing a corrected gene holds promise for all nations as researchers continue to refine and improve the therapy.

他补充表示，这一切不是突然发生的。此次试验投入了技术和成本，所以穷国的镰状细胞病患者不太可能进行尝试。但引入非病变基因的疗法为所有国家提供了希望，毕竟现在很多研究人员都在持续地完善和改进疗法。

1 sickle   

n.镰刀

参考例句：

• The gardener was swishing off the tops of weeds with a sickle.园丁正在用镰刀嗖嗖地割掉杂草的顶端。
• There is a picture of the sickle on the flag. 旗帜上有镰刀的图案。

2 anemia   

n.贫血，贫血症

参考例句：

• The doctors analyzed the blood sample for anemia. 医生们分析了贫血的血样。
• I was put on iron tablets for my anemia.我曾因贫血吃补铁药片。

3 suffocating   

a.使人窒息的

参考例句：

• After a few weeks with her parents, she felt she was suffocating.和父母呆了几个星期后，她感到自己毫无自由。
• That's better. I was suffocating in that cell of a room.这样好些了，我刚才在那个小房间里快闷死了。

4 karate   

n.空手道(日本的一种徒手武术)

参考例句：

• Alice's boyfriend knew a little karate.艾丽斯的男朋友懂一点儿空手道。
• The black belt is the highest level in karate.黑腰带级是空手道的最高级别。

5 vessels   

n.血管( vessel的名词复数 )；船；容器；（具有特殊品质或接受特殊品质的）人

参考例句：

• The river is navigable by vessels of up to 90 tons. 90 吨以下的船只可以从这条河通过。 来自《简明英汉词典》
• All modern vessels of any size are fitted with radar installations. 所有现代化船只都有雷达装置。 来自《现代汉英综合大词典》

6 clogging   

堵塞，闭合

参考例句：

• This process suffers mainly from clogging the membrane. 这种过程的主要问题是滤膜的堵塞。
• And you know that eyewitness that's been clogging up the airwaves? 你知道那个充斥着电视广播的目击证人？

7 defective   

adj.有毛病的，有问题的，有瑕疵的

参考例句：

• The firm had received bad publicity over a defective product. 该公司因为一件次品而受到媒体攻击。
• If the goods prove defective, the customer has the right to compensation. 如果货品证明有缺陷， 顾客有权索赔。

8 gene   

n.遗传因子，基因

参考例句：

• A single gene may have many effects.单一基因可能具有很多种效应。
• The targeting of gene therapy has been paid close attention.其中基因治疗的靶向性是值得密切关注的问题之一。

9 smears   

污迹( smear的名词复数 )； 污斑； （显微镜的）涂片； 诽谤

参考例句：

• His evidence was a blend of smears, half truths and downright lies. 他的证词里掺杂着诽谤、部份的事实和彻头彻尾的谎言。
• Anything written with a soft pencil smears easily. 用软铅笔写成的东西容易污成一片。