基因编辑治疗镰刀型贫血症首例
Victoria Gray slides open a closet door, pulls out a suitcase and starts packing piles of clothes.
维多利亚·格蕾推开了衣橱的门，取出一个行李箱，开始收拾自己的衣服。
"My goodness," says Gray.
“我的天哪，”格蕾说道。
"Did I really bring all this?"
“我真的要把这些都带上吗？”
Gray, who has sickle cell disease, is the first patient with a genetic disorder whom doctors in the United States have tried to treat using the powerful gene-editing technique CRISPR.
格蕾患有镰刀型红细胞症，在美国，她是第一个得到有效的基因编辑技术CRISPR治疗的患者。
Today, Gray is getting ready to finally go home to Forest, Miss., after months away from her four children so she could undergo the experimental treatment, which involves infusions of genetically modified bone marrow cells.
今天，格蕾终于可以准备回到密苏里州弗雷斯特市的家中，她为了接受实验性的治疗而离开孩子们已经数月了，这种治疗包括输入经过基因修饰的骨髓细胞。
"I'm very excited," says Gray, who has been living in a temporary apartment in Nashville, Tenn., for several weeks since getting discharged from the nearby TriStar Centennial Medical Center.
“我太开心了，”格蕾说道，在从附近的三星百年医疗中心出院后，她之前还在田纳西州纳什维尔市的一处临时公寓里住了几周。
"I know it's going to be emotional for me. I miss the hugs and the kisses and just everything."
“我想我肯定会很激动。我想念孩子们的拥抱和吻以及所有的一切。”
"Victoria is a pioneer in this. And we are very excited," says her doctor, Haydar Frangoul.
“维多利亚是这种治疗的先例。我们也感到很开心，”她的医生海达·弗兰格说道。
"I feel hopeful for the future," Gray says.
“我对未来感到充满了希望，”格蕾说道。
Sickle cell disease is a devastating genetic disease that afflicts millions of people around the world, including about 100,000 in the United States.
镰刀型贫血症是一种很严重的遗传性疾病，全球总共有上百万的患者，其中美国有约十万人左右。
The disease is caused by a genetic defect that turns red blood cells into hard, sticky, sickle-shaped cells that don't carry oxygen well, clog the bloodstream, damage organs and cause torturous bouts of pain.
这种病的成因是一种基因缺陷，使得红细胞变成了一种僵硬的，镰刀型的细胞，这些细胞携氧能力很差，会阻塞血流，损伤器官带来巨大的病痛。
"The pain is excruciating. It's like being in a car accident and having lightning in your chest. It's a pain that makes a grown woman like me scream," Gray says.
“这种痛太折磨人了。就好像在一次车祸中，胸腔被闪电击中一样。这样的痛会让哪怕像我这样的成年女子也哭出来，”格蕾说道。
"It's an overwhelming pain."
“这真是生命不能承受之痛。”
Like many sickle cell patients, Victoria had to drop out of school, quit work and spend weeks in the hospital away from her family.
跟很多镰刀型贫血症患者一样，维多利亚不得不中途辍学，辞职，并离开家人而花几周的时间去住院。
Since many sickle cell patients don't survive past their 40s, Gray worries whether she'll live to see her children grow up.
因为很多镰刀型贫血症患者都不能活过40岁，格蕾担心自己是否能看到自己的孩子们长大。
She just turned 34.
她今年刚刚34岁。
"It's horrible ... knowing that I could have a stroke or a heart attack ... at any time because I have these cells in me that are misshapen," Gray says.
“当得知因为我体内的细胞发生了异常，我可能随时会中风或者心脏病发作的时候，我感觉太可怕了。”
"Who wouldn't worry?"
“换做是谁都会担心的吧？”
But Gray has hope now, because in July doctors infused billions of her own bone marrow cells back into her body, after editing them with CRISPR.
但如今格蕾重获了希望，因为在七月，医生们将上亿个她自己的骨髓细胞基因编辑后重新把它们输入了她的体内。
Scientists used CRISPR to modify a gene in the cells to make them produce fetal hemoglobin, a protein that babies usually stop making shortly after birth.
科学家们利用CRISPR技术修饰了细胞中的基因使它们能够产生胎儿血红蛋白，这种蛋白通常在婴儿出生后很快就不再生产了。
The hope is that the protein produced through the gene-editing treatment will give sickle cell patients like Gray healthy red blood cells.
医生们希望这种通过基因编辑治疗生产的蛋白能够使像格蕾这样的镰刀型贫血症患者重新拥有健康的红细胞。
CRISPR enables scientists to make very precise changes in DNA, raising hopes that the technique will lead to new ways to prevent and treat many diseases.
CRISPR使得科学家们能够在DNA中进行精准的编辑，这项技术有望用于预防和治疗多种疾病。
The sickle cell study is part of a wave of studies that are moving CRISPR out of the lab and into the clinic.
对于镰刀型贫血症的研究就是这种技术潮流的一部分，并且正从实验室走向临床应用。
问题
文中提到的疾病是哪一种细胞出现了病变？
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