VOA常速英语2019--新研究：镰刀形贫血症或可治愈

（单词翻译）

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。