转基因皮肤挽救了一个危在旦夕的男孩,双语阅
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Genetically Altered Skin Saves A Boy Dying Of A Rare Disease-转基因皮肤挽回了一个危殆的男孩

In the future, we may not need to rely on human donations for life-saving skin grafts.

November 8, 20171:28 PM ET

前途,可弥补生命的肌肤移植大概没有要求再凭仗人类自己进献了。

Heard onAll Things Considered

That’s the goal of XenoTherapeutics, a Boston-based biotech nonprofit. Last week, the US Food and Drug Administration approved the group’s initial application for temporary skin grafts curated from genetically modified pigs. This means that they can start testing pig skin grafts on people who have experienced severe burns. It’s the first time that an animal organ has been cleared for human testing in the US.

Richard Harris

那正是分部设在罗马的非营利性生物科学技术公司塞诺移植医治公司的对象。下一周,U.S.食物和药物管理局批准了该公司利用基因查对猪的皮作为偶然移植皮肤的起来申请。这意味着,他们得以先河对严重久咳者举行猪皮移植测量检验。这也是美利坚合作国第1回批准用动物器官进行身体育项目检测试。

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Skin, the body’s largest organ, plays a crucial role in the immune system by blocking pathogens from reaching our vulnerable internal organs. It also holds in water, electrolytes, and other nutrients, and helps the body maintain a constant temperature. People with severe skin damage are at a high risk of developing deadly infections or organ failure as a result of changes in temperature or hydration.

Researchers

肌肤是人身最大的器官,可阻拦病原体侵入人体薄弱的体内器官,由此在免疫性系统中发挥珍视大的法力。别的,皮肤可锁住水分、电解质及其余三磷酸腺苷物质,还可扶持肉体保持体温平稳。皮肤严重受到伤害者因体温或水合状态的改变而患致命性感染或器官衰退的危机极高。

grew sheets of genetically altered skin cells in the lab and used them

Skin grafts can help protect these patients as they heal. At the moment, the only skin grafts available in the US come from cadavers who have agreed to be organ donors, or patients who have undergone surgery to remove excess skin after dramatic weight loss. These human skin used for grafts are a “rare commodity.”

to treat a boy withlife-threatening epidermolysis bullosa.

移植皮肤可在伤者康复进程中对伤者加以护卫。当前,在美利坚独资国得到移植皮肤的独一渠道是利用同意贡献器官者死后留下的皮肤,或因体重严重下跌做多余皮肤去除术的病人皮肤。用于移植的人类皮肤可谓是“稀缺商品”。

研商人口在实验室中中构建转基因皮肤细胞,用它们来治得了疗危及人命的大疱性表皮松解症的男孩。

XenoTherapeutics, which gets its name from xenotransplantation, or animal-to-human transplants, has bred pigs that have skin remarkably similar to our own. Although pig skin normally produces a type of sugar human skin does not, these pigs have been genetically modified not to make it. Grafts from these pigs are therefore more likely to slide under the radar of the host’s immune system—at least temporarily. The idea is that they could be used for immediate burn treatment, followed human skin graft treatment later.

CMR Unimore/Nature

塞诺移植医治公司的爱沙尼亚语名就源自异种器官移植概念——即把动物器官移植给身体。该商厦喂养的猪,其猪皮与我们人类的皮肤极为经常。猪皮日常会生成一种糖而人皮则不会,为了使那一个猪不生成这种糖,该公司对那一个猪执行了基因校正。那样一来,那一个猪皮通过接受移植者免疫性系统监测的大概就更加高了——起码长时间内足以透过。那么些猪皮可被用于急切健忘管理,之后再拓宽人皮移植。

A

“I would venture that if we did a Coke and Pepsi side-by-side comparison… you’d be hard-pressed to tell which was the human cadaveric allograft versus [the pig graft],” said XenoTherapeutics CEO Paul Holzer.

child who was on the verge of death from a rare inherited disease has

塞诺移植临床集团CEOPaul·霍尔泽说:“笔者敢说,那就像您把七喜和七喜放在一块儿展开比较,你很难分得清哪个是根源人类尸体的同种异体移植皮肤,哪个是移植猪皮。”

been treated with genetically engineered skin cells that replaced most

The nonprofit has been working closely with physicians at Massachusetts General Hospital, who will help conduct the clinical trials starting next month. The first trial will only be testing the grafts’ safety and tolerability in six patients with severe burns. Assuming the results are positive after a month, the grafts will need to undergo two more stages of testing before they can be approved for widespread clinical use.

of the skin on his body.

这家非营利性集团直接在与亚拉巴马综合医院的医务职员们展开紧凑合营,那所医院将从前段时间起扶持进行临床实验。第一回临床实验将只在6名严重烧病人身上测验移植的安全性以致耐受性。借使一个月后临床实验结果是积极的,移植专业还要开展四个级次的测量检验,而后才可获批推广来临床使用。

三个因少有遗传病痛将近长逝的子女正在用转基因皮肤细胞代替了自己超越55%的皮肤。

Several other groups around the world are working to make animal organs suitable for clinical medicine. In Brazil, researchers are exploring using tilapia skin for use as temporary bandages for burn victims whose skin is regrowing. Just last week, scientists from Germany reported that they had made a crucial step in keeping baboons given genetically modified pig hearts alive for half a year. Their success suggests that pig hearts could one day be used to treat patients with heart failure.

The

除塞诺移植临床集团外,目后天下还恐怕有多少个团体在商讨将动物器官适用于临床工学。举例在巴西联邦共和国,研商人口正在研商用非洲鲫壳子的鱼皮作为正在重新长出皮肤的烧病者的一时半刻绑带。而就在下二十日,多位德意志地医学家告诉称他们也迈出关键一步,成功地让接受基因更正猪心脏移植的狒狒存活了四个月岁月。那申明猪心脏以往将或许被用来医疗心衰病人。

treatment represents a notable success for the field of genetherapy,

which has suffered manysetbacks. And it's potentially good news for

children suffering from a painful and often deadly skin condition called

epidermolysis bullosa.

固然经历了累累为山止篑,但该疗法在基因医治领域依旧赢得了综上可得的成功,对于患有大疱性表皮松解症这种疼痛且有时致命皮肤病的儿童来讲,这可能是个好音讯。

In

this disease, children are born with a flawed gene that prevents the

outer layer of the skin, theepidermis, from binding to the inner layer.

This can cause excruciatingblistersto form all over these children's

bodies.

在这里种病痛中,小孩子先性子就患有一个基因缺欠,他拦挡皮肤、表皮的外围与内层结合。那会在小孩的浑身引起水泡。

In

the case in Europe, a 7-year old boy ended up in the hospital back in

2015 after 60 percent of his epidermis had sloughed off. Tobias

Rothoeft, a surgeon at a burn unit at Ruhr University in Bochum,

Germany, says he and his colleagues tried everything — including a skin

transplant from the boy's father —to no avail.

在澳大澳门联邦(Commonwealth of Australia)的贰个病例,一个7岁的男孩住进了卫生院,二零一四年后他的四分之一的肌肤已经脱落。TobiasRothoeft,在波鸿的鲁尔大学的一名黄疸科医务职员德意志联邦共和国说,他和他的同事们品尝了全套——满含男孩阿爹的肌肤移植——但都不行。

"After nearly two months we were absolutely sure there was nothing we could do for this kid and that he would die," Rothoeft said in a telephone news conference hosted byNature, which publishedthe studyonline Wednesday.

“在通过近八个月大家相对信赖大家帮不了那个孩子,他会死的,”Rothoeft在《自然》杂志的电话情报宣布会上说,这段话在周二也被公布到了互联网上。

Rothoeft and his colleagues took one last look around the medical literature and learned of researchers in Italy who were experimenting with anew treatment00126-4)for this disease. Michele De Luca and colleagues at the University of Modena and Reggio Emilia were genetically engineering skin cells to repair the inborn flaw.

Rothoeft和她的同事们不常见到了一份医学文献,获悉意大利共和国的钻探人口正在考试治疗这种病痛的新的看病措施。来自意大利共和国摩德纳大学Michele De Luca和Cole正选取基因工程的肌肤细胞修复基因的原状缺欠。

De

Luca used a virus toinserta healthy gene into cells taken from the

boy's skin. Some of those cells,stem cells,multiply indefinitely. So De

Luca was able to grow entire sheets of engineered epidermis, which were

shipped to the hospital in Germany.

De Luca用一种病毒将常规的基因插入从男孩皮肤中抽出的细胞中。在那之中一些细胞,干细胞,无有效期繁衍。所以De 卢卡能够一整面包车型大巴转基因的人工表皮,它被运往了德意志联邦共和国的卫生院。

De

Luca had used this procedure successfully in 2006 to replace a

relatively small patch of skin on another patient. But this boy needed

to have 80 percent of his skin replaced with grafts of this genetically

modified material. It took two operations, both in the fall of 2015.

De Luca在二〇〇六年成事地利用了那个办法来替换另一个患儿的一小块皮肤。不过这么些男孩必要百分之八十的皮肤移植。在二〇一四年秋日,他累积举行了四遍手术。

"In

the first one, we grafted all four limbs," De Luca told reporters in

the press call. "In the second operation we grafted the remaining part

of the body, mainly the back."

De Luca在收受新闻报道工作者搜求时对媒体人说:“第一遍,我们移植了具有四肢的皮层。”在第三遍手术中,我们移植了身体的多余部分,首假设背部。

After

eight months in the intensive care unit, the boy was well enough to go

home. And, two years later, he is in school, even playing soccer.

在ICU呆了3个月后,那个男孩已经痊愈了,他能够回家了。六年后,他上学了,以至足以踢足球。

"The

kid is doing quite well," Rothoeft said. "The skin is of good quality,

it doesn't need any ointments or stuff like that. It's perfectly smooth

and it is quite stable. And if he gets any bruises, they

justheallikebruisesin every other kid."

“这么些孩子复苏得很好,”Rothoeft说。“皮肤的成色是好的,它无需别的药膏或类似的事物。它不行平整,並且一定平稳。如若他有瘀伤,他们就如别的子女的瘀伤同样会融洽愈合。”

Onelingeringquestion is the concern that gene therapies like this, involving viruses, can increase the risk of cancer. That's because the viruses insert the new gene randomly into human DNA. Anattemptat using gene therapy to treat severe combined immuneodeficiency (SCID) in 2002 ended up triggering cancer in some patients.

一个挥之不去的标题是,这种基因疗法,包罗病毒,或然会加多患有恶性肿瘤症的危机。这是因为病毒将新基因随机地插入人类的DNA中。二零零三年,在叁遍尝试用基因疗法医疗重症联合性免疫破绽(SCID)的时候,所吸引的癌症让一名换着住进了医院。

That bad result set back the field of gene therapy, though there have since been successes treatingSCID, and most recently, cancer.

那一遍于结果阻碍了基因医疗领域的迈入,固然新兴已经成功地医疗了SCID和近来的癌症。

"Certainly

it is a potential problem," De Luca said. But he generated hundreds of

millions of cells during this procedure and didn't see anything of

concern. And in this case, clearly the benefits of treating the boy

outweighed the risks.

“当然,那是一个暧昧的主题素材,”De Luca说。但她在这里个进度中发生了上亿的皮层细胞,并不曾发出别的让人挂念的职业。在此种气象下,显著医疗男孩的好处是远远不独有风险的。

News of this is just starting to trickle out to advocates who have children with epidermolysis bullosa.

这一新闻正是被一名患有大疱性表皮松解症的子女的父母发表的。

"I think it's groundbreaking," says Brett Kopelan, who heads a U.S. organization focused on this disease, known by its acronym,debra. "I think it's incredibly exciting."

“作者以为那是开创性的,”关切那个毛病美利哥组织的领导职员,BrettKopelan说,“作者以为那不行令人高兴。”

His 10-year-old daughter Rafi has a severe case.

他10岁的丫头Rafi患有这种病魔。

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Rafi

Kopelan, 10, has epidermolysis bullosa, which causes painful blisters

of the skin and mucous membranes. Her father, Brett, is at right.

Rafi Kopelan,10岁,患有大疱性表皮松解症,导致忧伤的水泡性皮肤和粘膜。她的阿爹,Brett,在右边。

Courtesy of Brett Kopelan

"Imagine

the last time you had a paper cut and you put some Purell on it and it

stung, right?" Kopelan says. "Now imagine that being 60 percent of your

body.

“想象一下,你上三遍被纸割到,你用某些消毒液来冲洗,渐渐有一些刺痛,对吧?”Kopelan说。”将来想像一下,如果您肉体的十分三的皮肤都经历这种处境的时候。

"When

you have to do a bath and bandage change every day, you are subjected

to severe torture. It's incredibly painful, and it can take up to 3 to 4

hours a day," he says. "As a parent, there's not a day that goes by

that a little bit of my heart doesn't break.

当您每一日必须洗澡和换绷带时,你会蒙受沉痛的折腾。这是令人匪夷所思的悲苦,每一天,它大概供给长达3至4钟头,”他说。作为家长,每时每刻,作者都是零散不已。

"Yet

Rafi, knowing that it's going to be an incredibly painful couple of

hours, walks into the bath and bandage room ... so that we can clean her

wounds to make sure they don't get infected and to prevent a

potentially life-threatening situation.

然而,Rafi,知道那将是悲苦的几钟头,走进浴室和绷带的屋企…,我们能够清理她的创口,幸免感染和防御可能危及人命的事态。

"She's the bravest person I know."

“她是个铁汉的姑娘”

In

addition to the painfulblisters, Rafi needs frequent throat surgeries,

because her condition also affects mucous membranes. She often uses a

wheelchair because it's so painful to walk.

除开难受的水沫,Rafi须求频仍喉腔手术,因为他的病状也耳闻则诵粘膜。她时常坐轮椅,因为走路太优伤了。

"My

daughter would love to be able to not have to wear bandages on a daily

basis, she'd love to jump in a pool without worrying about it hurting,

or taking a shower — or even wearing shoes."

“作者闺女很想天天都能够不用带绷带,她喜欢在游泳池里跳,不用操心会受到损伤,恐怕洗浴,以至穿鞋。”

The skin therapy described in theNaturepaper wouldn't cure her — in fact, it targets a different genetic defect that causes the same condition. But a similar approach could reduce the agony of daily living. And Kopelan says medical interest in this disease is now growing rapidly.

《自然》描述的皮层疗法不能够真正治愈她——事实上,它针对的是一种导致同样情状的差异的遗传破绽。不过类似的不二秘诀能够减去每日生活上的惨重。Kopelan说,这种病如今正麻利引起历史学界的野趣。

"We've

gone from zero biotechnology and pharmaceutical [companies] to like 12

companies, so we're really at aninflectionpoint right now," he says.

“大家早已从0家生物本领和制药[公司]提升到了12家公司,所以我们今后正处在五个拐点,”他说。

Experimentaltreatments are getting under way in the United States and Asia, as well as in Europe. Peter Marinkovich, Jean Tang and colleagues at the Stanford University School of Medicine areusing the same approachas De Luca, and they have treated seven children using smaller patches of skin.

U.S.A.和南美洲以致亚洲正值扩充试验性治疗。Peter 马林kovich,姬恩Tang和她的同事们在巴黎综合理历史高校经济高校使用和De Luca同样的点子,用小块皮肤医疗了四个儿女。

Marinkovich

tells Shots that their long-term goal is to treat a child's entire

body, and the research is gradually laying the groundwork to do that.

The severely injured child in Germany offered a unique opportunity to

try that, and the encouraging results are generating more enthusiasm. "I

was super impressed when I saw [the] results," Marinkovich says.

马林kovich告诉媒体人,他们的长时间目的是医治男女的一体身子,和以稳步的研商做铺垫。德意志受杀害的有的小孩提供了三个品尝的机缘,令人鼓劲的结果会激情更加多的古道热肠。当本人看见结果后给自个儿留下了精品浓郁的记念,”马林kovich说。

“本译文仅供个人研习、欣赏语言之用,谢绝任何转载及用于别的商业用途。本译文所涉法律后果均由本身肩负。本身同意简书平台在接获有关作品权人的布告后,删除小说。”

原稿来自  NP汉兰达Richard HarrisNovember 8, 20171:28 PM ET

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