双语新闻：健康专家宣布,一种治疗镰状细胞病的新基因编辑疗法对患者是安全的

A group of health experts recently announced that a new gene editing treatment for sickle cell disease was safe for patients. The treatment could provide a new cure that attacks the disease at its genetic source.

一组健康专家最近宣布，一种治疗镰状细胞病的新基因编辑疗法对患者是安全的。这种疗法可能会提供一种新的治疗方法，从基因源头攻击这种疾病。

Sickle cell disease (SCD) is a genetic blood disorder that affects millions of people worldwide. The disease is common in places with high cases of malaria, like Africa and India. It strikes among Black Americans, Africans, and Middle Easterners more than other groups. Scientists believe being a carrier of SCD helps protect against severe malaria.

镰状细胞病(SCD)是一种遗传性血液疾病，影响着全世界数百万人。这种疾病在疟疾高发地区很常见，比如非洲和印度。美国黑人、非洲人和中东人比其他群体更容易患此病。科学家认为，作为SCD的携带者有助于预防严重的疟疾。

People with SCD carry unhealthy blood cells that result in blockages, called blood clots. This can cause pain and damage bones and organs in the human body. 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.

患有SCD的人携带不健康的血细胞，导致阻塞，称为血凝块。这会引起疼痛，损害人体的骨骼和器官。世界卫生组织表示，许多患有这种疾病最严重形式的儿童在五岁之前死亡，通常死于感染或严重失血。

Vertex Pharmaceuticals and CRISPR Therapeutics make the new treatment, called "exa-cel." The treatment involves permanently changing the genetic material, DNA, in a patient's blood cells. The goal is to help the body return to healthy blood cell production. Healthy blood cells are present at birth but abnormal cells begin to develop in people with sickle cell disease.

顶点制药公司和CRISPR治疗公司制造了这种名为“exa-cel”的新疗法。这种治疗方法包括永久改变患者血细胞中的遗传物质DNA。目标是帮助身体恢复健康的血细胞生产。健康的血细胞在出生时就存在，但镰状细胞病患者开始出现异常细胞。

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. Patients get medicines to kill off other damaged blood-producing cells and then are given back their changed stem cells.

当患者接受治疗时，从他们的血液中取出干细胞，并使用基因编辑工具CRISPR去除开关基因。患者服用药物来杀死其他受损的造血细胞，然后再将改变后的干细胞放回原位。

Vertex reported 46 people got the treatment in its study. Among 30 of them who had at least 18 months of observation, 29 were free of pain crises for at least a year. All 30, Vertex reported, avoided being hospitalized for pain crises for that long.

Vertex报告说，在研究中有46人接受了这种治疗。在30名至少观察了18个月的患者中，29名患者至少一年没有出现疼痛危机。据Vertex报道，所有30名患者都避免因疼痛危机住院治疗这么长时间。

Victoria Gray, of Mississippi, was the first patient to test the "exa-cel" treatment. Gray told researchers at a scientific gathering earlier this year that she had suffered from pain since childhood. She described feeling "reborn" the day she got the treatment. "My children no longer have a fear of losing their mom to sickle cell disease," she said.

密西西比州的维多利亚·格雷是第一位测试这种“exa-cel”疗法的患者。格雷在今年早些时候的一次科学会议上告诉研究人员，她从小就饱受疼痛之苦。她描述了接受治疗的那天感觉“重生”。她说:“我的孩子们再也不用担心妈妈会因为镰状细胞病而去世。”

The U.S. Food and Drug Administration (FDA) had already found the treatment effective in a study of SCD patients. 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.

美国食品和药物管理局(FDA)已经在一项针对SCD患者的研究中发现这种治疗方法是有效的。然而，卫生机构不确定研究中使用的小基因样本是否涵盖了整个美国镰状细胞病人群。

With the experts' findings, the FDA is likely to approve the treatment in early December for patients age 12 and older. If the treatment is approved, Vertex has also proposed to follow up with patients to study its safety for another 15 years.

根据专家的研究结果，FDA可能会在12月初批准对12岁及以上患者的治疗。如果该疗法获得批准，Vertex还提议对患者进行随访，再进行15年的安全性研究。

Vertex has not said how much the treatment will cost.

Vertex尚未透露治疗费用。

But the Institute for Clinical and Economic Review estimates the cost could be around $2 million for the treatment. 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.

但临床和经济评论研究所估计，这种治疗的成本可能在200万美元左右。相比之下，今年早些时候的研究显示，从出生到65岁，镰状细胞治疗的医疗费用总计可能高达170万美元。

Dr. Allison King of Washington University School of Medicine in St. Louis, Missouri cares for children and young adults with sickle cell disease. She told the Associated Press that the new treatments would be costly.

位于密苏里州圣路易斯市的华盛顿大学医学院的Allison King博士负责治疗患有镰状细胞病的儿童和年轻人。她告诉美联社，新的治疗方法将是昂贵的。

"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?"

“但如果你仔细想想，”她说，“让一个人感觉好一点，不再痛苦，不再一直呆在医院里，这值多少钱?”