在經(jīng)歷了病痛后，病人希望基因編輯能有所幫助

在經(jīng)歷了病痛后，病人希望基因編輯能有所幫助

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.

維多利亞·格雷收拾行囊準(zhǔn)備離開田納西州的納什維爾。她是第一個參與使用CRISPR治療鐮狀細(xì)胞病試驗的病人。

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

維多利亞·格雷打開壁櫥的門，拿出一個手提箱，開始打包衣服。

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

“我的天啊，”格雷說。“我真的要把這些全帶走嗎?”

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.

格雷患有鐮狀細(xì)胞病，是美國醫(yī)生嘗試使用強(qiáng)大的基因編輯技術(shù)CRISPR治療的第一個遺傳疾病患者。

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，格雷在離開四個孩子幾個月后，終于準(zhǔn)備回到密西西比州福里斯特的家中，以便進(jìn)行實(shí)驗性治療。

"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."

“我很興奮，”格雷說，她一直住在田納西州納什維爾的一間臨時公寓里。從附近的三星百年醫(yī)療中心出院后，她已經(jīng)住了幾個星期。“我知道這會讓我很激動。我想念那些擁抱、親吻。”

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

美國國家公共電臺獨(dú)家報道了格雷的臨床試驗過程。

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

“維多利亞是這方面的先行者。我們非常興奮，”她的醫(yī)生海達(dá)爾·弗朗古說。

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

“我對未來充滿希望，”格雷說。

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

鐮狀細(xì)胞病是一種毀滅性的遺傳疾病，困擾著全世界數(shù)百萬人，其中約有10萬美國人。

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.

這種疾病是由一種遺傳缺陷引起的，這種遺傳缺陷會使紅細(xì)胞變成堅硬的、粘性的、鐮刀狀的細(xì)胞，這些細(xì)胞不能很好地輸送氧氣，阻塞血流，損害器官并引起劇烈的陣痛。

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

“疼痛難忍。這就像遭遇了一場車禍，胸口有閃電一樣。”格雷說。

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.

像許多鐮狀細(xì)胞病人一樣，維多利亞不得不輟學(xué)，辭掉工作，離開家人在醫(yī)院呆上幾周。由于許多鐮狀細(xì)胞病人不能活過40歲，格雷擔(dān)心她能否活到孩子長大。她剛滿34歲。

"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?"

格雷說:“知道自己可能在任何時候中風(fēng)或心臟病發(fā)作，因為我體內(nèi)的這些細(xì)胞是畸形的，這太可怕了。”“誰不擔(dān)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.

但是格雷現(xiàn)在有了希望，因為在7月份，醫(yī)生們用CRISPR對她的骨髓細(xì)胞進(jìn)行了編輯，然后將數(shù)十億的骨髓細(xì)胞注入她的體內(nèi)。

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.

今年晚些時候，波士頓的醫(yī)生們計劃使用CRISPR編輯患者視網(wǎng)膜中的細(xì)胞，希望能恢復(fù)遺傳性失明患者的視力。

Gray calls her edited cells her "super cells."

格雷稱她的被編輯過的細(xì)胞為“超級細(xì)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.

格雷說:“它們必須非常棒，才能對我的身體產(chǎn)生影響，幫助我變得更好，讓我有更多的時間陪伴我的孩子和家人。”

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

她知道現(xiàn)在還不清楚什么時候她才能知道它是否起作用。

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

最困難的時候已經(jīng)過去了。所以現(xiàn)在只需要等待。”