在经历了病痛后，病人希望基因编辑能有所帮助

在经历了病痛后，病人希望基因编辑能有所帮助

Victoria Gray packs her bags in preparation for leaving Nashville, Tenn., where she has been the first patient involved in a trial using CRISPR to try to treat sickle cell disease.

维多利亚·格雷收拾行囊准备离开田纳西州的纳什维尔。她是第一个参与使用CRISPR治疗镰状细胞病试验的病人。

Victoria Gray slides open a closet door, pulls out a suitcase and starts packing piles of clothes.

维多利亚·格雷打开壁橱的门，拿出一个手提箱，开始打包衣服。

"My goodness," says Gray. "Did I really bring all this?"

“我的天啊，”格雷说。“我真的要把这些全带走吗?”

Gray, who has sickle cell disease, is the first patient with a genetic disorder who doctors in the United States have tried to treat using the powerful gene-editing technique CRISPR.

格雷患有镰状细胞病，是美国医生尝试使用强大的基因编辑技术CRISPR治疗的第一个遗传疾病患者。

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.

今天，格雷在离开四个孩子几个月后，终于准备回到密西西比州福里斯特的家中，以便进行实验性治疗。

"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. "I know it's going to be emotional for me. I miss the hugs and the kisses."

“我很兴奋，”格雷说，她一直住在田纳西州纳什维尔的一间临时公寓里。从附近的三星百年医疗中心出院后，她已经住了几个星期。“我知道这会让我很激动。我想念那些拥抱、亲吻。”

NPR has exclusive access to chronicle Gray's journey through the highly anticipatedclinical trial.

美国国家公共电台独家报道了格雷的临床试验过程。

"Victoria is a pioneer in this. And we are very excited," says her doctor, Haydar Frangoul.

“维多利亚是这方面的先行者。我们非常兴奋，”她的医生海达尔·弗朗古说。

"I feel hopeful for the future," Gray says.

“我对未来充满希望，”格雷说。

Sickle cell disease is a devastating genetic disease that afflicts millions of people around the world, including about 100,000 in the United States.

镰状细胞病是一种毁灭性的遗传疾病，困扰着全世界数百万人，其中约有10万美国人。

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 tortuous bouts of pain.

这种疾病是由一种遗传缺陷引起的，这种遗传缺陷会使红细胞变成坚硬的、粘性的、镰刀状的细胞，这些细胞不能很好地输送氧气，阻塞血流，损害器官并引起剧烈的阵痛。

"The pain is excruciating. It's like being in a car accident and having lightening in your chest. " Gray says.

“疼痛难忍。这就像遭遇了一场车祸，胸口有闪电一样。”格雷说。

Like many sickle cell patients, Victoria had to drop out of school, quit work and spend weeks in the hospital away from her family. Since many sickle cell patients don't survive past their 40s, Gray worries whether she'll live to see her children grow up. She just turned 34.

像许多镰状细胞病人一样，维多利亚不得不辍学，辞掉工作，离开家人在医院呆上几周。由于许多镰状细胞病人不能活过40岁，格雷担心她能否活到孩子长大。她刚满34岁。

"It's horrible — knowing that I could have a stroke or a heart attack or at any time because I have these cells in me that are misshapen," Gray says. "Who wouldn't worry?"

格雷说:“知道自己可能在任何时候中风或心脏病发作，因为我体内的这些细胞是畸形的，这太可怕了。”“谁不担心呢?”

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.

但是格雷现在有了希望，因为在7月份，医生们用CRISPR对她的骨髓细胞进行了编辑，然后将数十亿的骨髓细胞注入她的体内。

Later this year, doctors in Boston are planning to use CRISPR to edit cells in patients' retinas, in hopes of restoring vision in patients with an inherited form of blindness.

今年晚些时候，波士顿的医生们计划使用CRISPR编辑患者视网膜中的细胞，希望能恢复遗传性失明患者的视力。

Gray calls her edited cells her "super cells."

格雷称她的被编辑过的细胞为“超级细胞”。

"They gotta be super to do great things in my body and to help me be better and help me have more time with my kids and my family," Gray says.

格雷说:“它们必须非常棒，才能对我的身体产生影响，帮助我变得更好，让我有更多的时间陪伴我的孩子和家人。”

She knows it's unclear when she will know whether it's working.

她知道现在还不清楚什么时候她才能知道它是否起作用。

The hardest part is over. So now it's just waiting."

最困难的时候已经过去了。所以现在只需要等待。”