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A group of health experts recently announced that a new gene editing treatment for sickle cell disease was safe for patients.
一個健康專家小組最近宣布，一種新的針對鐮狀細胞病的基因編輯療法對患者來說是安全的。
The treatment could provide a new cure that attacks the disease at its genetic source.
這種療法可能會提供一種新的從基因源頭攻克這種疾病的治愈方法。
Sickle cell disease (SCD) is a genetic blood disorder that affects millions of people worldwide.
鐮狀細胞病是一種遺傳性血液疾病，影響全球數百萬人。
The disease is common in places with high cases of malaria, like Africa and India.
這種疾病在瘧疾高發區很常見，比如非洲和印度。
It strikes among Black Americans, Africans, and Middle Easterners more than other groups.
相較于其他群體，美國黑人、非洲人和中東人更容易患鐮狀細胞病。
Scientists believe being a carrier of SCD helps protect against severe malaria.
科學家認為，成為鐮狀細胞病的攜帶者有助于預防嚴重的瘧疾。
People with SCD carry unhealthy blood cells that result in blockages, called blood clots.
患有鐮狀細胞病的人攜帶著不健康的血細胞，它們會導致阻塞，即血栓。
This can cause pain and damage bones and organs in the human body.
這會引起疼痛，損害人體的骨骼和器官。
The World Health Organization said many children with the most severe form of the disease die before the age of five, usually from an infection or severe blood loss.
世界衛生組織表示，許多患有最嚴重鐮狀細胞病的兒童在五歲之前死亡，通常死于感染或嚴重失血。
Vertex Pharmaceuticals and CRISPR Therapeutics make the new treatment, called "exa-cel."
福泰制藥和CRISPR Therapeutics公司創造了這種名為“exa-cel”的新療法。
The treatment involves permanently changing the genetic material, DNA, in a patient's blood cells.
這種療法包括永久改變患者血細胞中的遺傳物質DNA。
The goal is to help the body return to healthy blood cell production.
其目標是幫助身體恢復健康的血細胞生產功能。
Healthy blood cells are present at birth but abnormal cells begin to develop in people with sickle cell disease.
健康的血細胞在出生時就存在，但患有鐮狀細胞病的人開始出現異常細胞。
When patients receive the treatment, stem cells are removed from their blood and the gene-editing tool CRISPR is used to remove the switching gene.
當患者接受治療時，醫生會從他們的血液中移除干細胞，并使用基因編輯工具CRISPR移除開關基因。
Patients get medicines to kill off other damaged blood-producing cells and then are given back their changed stem cells.
患者服用藥物來殺死其他受損的造血細胞，然后醫生再把編輯過的干細胞放回原位。
Vertex reported 46 people got the treatment in its study.
福泰制藥報告稱，有46人在其研究中接受了這種治療。
Among 30 of them who had at least 18 months of observation, 29 were free of pain crises for at least a year.
在30名至少被觀察了18個月的患者中，有29名患者至少一年沒有出現疼痛危機。
All 30, Vertex reported, avoided being hospitalized for pain crises for that long.
據福泰制藥報告，所有30名患者在很長一段時間內都沒有因疼痛危機而住院。
Victoria Gray, of Mississippi, was the first patient to test the "exa-cel" treatment.
密西西比州的維多利亞·格雷是第一個測試這種“exa-cel”療法的患者。
Gray told researchers at a scientific gathering earlier this year that she had suffered from pain since childhood.
格雷在今年早些時候的一次科學會議上告訴研究人員，她從小就飽受疼痛之苦。
She described feeling "reborn" the day she got the treatment.
她稱，在接受治療的那一天，她感覺自己“重獲新生”。
"My children no longer have a fear of losing their mom to sickle cell disease," she said.
她說：“我的孩子們再也不用害怕因為鐮狀細胞病而失去他們的母親”。
The U.S. Food and Drug Administration (FDA) had already found the treatment effective in a study of SCD patients.
美國食品藥品監督管理局已經在一項針對鐮狀細胞病患者的研究中發現了這種治療方法的有效性。
The health agency, however, was not sure if the small genetic sample size used in the study captured the entire U.S. population for sickle cell disease.
然而，該衛生機構不確定研究中使用的少量基因樣本是否涵蓋了整個美國鐮狀細胞病患者群。
With the experts' findings, the FDA is likely to approve the treatment in early December for patients age 12 and older.
根據專家的研究結果，美國食品藥品監督管理局可能會在12月初批準對12歲及以上患者使用這種療法。
If the treatment is approved, Vertex has also proposed to follow up with patients to study its safety for another 15 years.
如果該療法獲得批準，福泰制藥還提議對患者進行隨訪，再進行15年的安全性研究。
Vertex has not said how much the treatment will cost.
福泰制藥尚未透露治療費用。
But the Institute for Clinical and Economic Review estimates the cost could be around $2 million for the treatment.
但臨床和經濟審查研究所估計，這種療法的費用可能在200萬美元左右。
By comparison, research earlier this year showed medical costs for sickle cell treatments, from birth to age 65, could add up to $1.7 million.
相比之下，今年早些時候的研究顯示，鐮狀細胞病患者從出生到65歲的醫療費用總計可能高達170萬美元。
Dr. Allison King of Washington University School of Medicine in St. Louis, Missouri cares for children and young adults with sickle cell disease.
密蘇里州圣路易斯市華盛頓大學醫學院的艾利森·金博士負責治療患有鐮狀細胞病的兒童和年輕人。
She told the Associated Press that the new treatments would be costly.
她告訴美聯社，新的治療方法費用高昂。
"But if you think about it," she said, "how much is it worth for someone to feel better and not be in pain and not be in the hospital all the time?"
她說：“但如果你仔細想想，讓人感覺好一些，不再痛苦，不再一直待在醫院里，這值多少錢？”
I'm Caty Weaver.
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