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实验疗法拯救了患有罕见疾病的男孩

时间:2017-12-13 14:26来源:互联网 提供网友:nan   字体: [ ]
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Experimental Therapy Saves Boy With Rare Disease

From VOA Learning English, this is the Health & Lifestyle report.

It is one of the worst things a parent can imagine.

Your child is sick, and suffering in terrible pain. No medical treatments seem to help.

That was the condition of a 9-year-old boy with an incurable1 skin disease. The father says his son was near death.

"He was in severe pain and asking a lot of questions I could not answer: 'Why do I suffer from this disease? Why do I have to live this life?'"

Today, the boy is no longer suffering like that. In fact, he is healthy and said to be living a normal life. His recovery shows what gene2 therapy can do.

This boy, whose parents do not want him identified, had until recently been suffering from a rare genetic3 skin condition.

The disease is called junctional epidermolysis bullosa. It causes painful wounds and blisters5 all over the body. The skin is so fragile that it tears and damages easily.

The child’s doctors in Germany said that none of their treatments was working. Left with no other choice, they said they simply wanted to ease the child’s pain and suffering as much as possible.

However, the boy’s parents did not accept that. They begged the doctors to look for experimental treatments.

One of the German doctors is Tobias Rothoeft at Ruhr University in Germany.

"So then we thought we could do nothing for this kid and opted7 for a palliative care approach. We discussed it with the family, and they begged us to do something about it or to try anything we could."

The boy’s father researched gene therapy on the internet. He told the doctors about his findings. They had never dealt with genetic therapy. The parents urged the doctors to consider the experimental treatment. When the doctors saw that experiments were being carried out, they reached out to another medical expert.

"Then we got in contact with Professor De Luca and we discussed this case, and he offered us to give us skin to cover the whole body of this kid."

Michele De Luca works with the Center for Regenerative Medicine in Modena, Italy. De Luca says the German doctors tried everything they could to help the boy. But the doctors had few real choices.

The German doctors asked De Luca if he and his team could try the genetic treatment on the patient. They called it "an act of compassion8."

De Luca explained gene therapy was still experimental. However, he agreed to try it as a treatment of last resort for the boy.

De Luca's team used a virus to put a healthy gene into cells taken from the boy’s skin. Some of those cells can multiply indefinitely. So, De Luca was able to grow new, healthy skin for the boy which he then sent to doctors in Germany. The German doctors used this genetically9 engineered skin to replace most of the skin on the child’s body.

De Luca says the child appeared better in October after the first transplant on the four limbs. The second operation was on his back and was a bigger transplant.

This is when doctors saw that the regeneration, or restoration, of the skin did something to the boy. He got better right away. The child's vital signs, such as body temperature and blood pressure, began getting better. That is when the doctors realized that the operation was a success.

Again, here is one of the boy’s German doctors, Tobias Rothoeft.

"We're happy that it worked. This kid is back to his normal life again. That's what we dreamed of doing and it was possible."

Two years later, the boy is now back in school, and no longer takes special medication. However, his doctors will have to watch him closely over time to watch for skin cancer or other problems.

For the Health & Lifestyle report, I'm Anna Matteo.

Words in This Story

gene therapy – medical n. the transplantation of normal genes10 into cells in place of missing or defective11 ones in order to correct genetic disorders12.

blister4 – n. a raised area on the skin that contains clear liquid and that is caused by injury to the skin

fragile – adj. easily broken or damaged

opt6 – v. to make a choice; especially : to decide in favor of something

palliative – medical n. : something that reduces the effects or symptoms of a medical condition without curing it

compassion – n. a feeling of wanting to help someone who is sick, hungry, in trouble, etc.

last resort – n. something done only if nothing else works

indefinitely – adv.

transplant – medical : to perform a medical operation in which an organ or other part that has been removed from the body of one person is put into the body of another person

vital signs – n. important body functions (such as breathing and heartbeat) that are measured to see if someone is alive or healthy


点击收听单词发音收听单词发音  

1 incurable incurable     
adj.不能医治的,不能矫正的,无救的;n.不治的病人,无救的人
参考例句:
  • All three babies were born with an incurable heart condition.三个婴儿都有不可治瘉的先天性心脏病。
  • He has an incurable and widespread nepotism.他们有不可救药的,到处蔓延的裙带主义。
2 gene WgKxx     
n.遗传因子,基因
参考例句:
  • A single gene may have many effects.单一基因可能具有很多种效应。
  • The targeting of gene therapy has been paid close attention.其中基因治疗的靶向性是值得密切关注的问题之一。
3 genetic PgIxp     
adj.遗传的,遗传学的
参考例句:
  • It's very difficult to treat genetic diseases.遗传性疾病治疗起来很困难。
  • Each daughter cell can receive a full complement of the genetic information.每个子细胞可以收到遗传信息的一个完全补偿物。
4 blister otwz3     
n.水疱;(油漆等的)气泡;v.(使)起泡
参考例句:
  • I got a huge blister on my foot and I couldn't run any farther.我脚上长了一个大水泡,没办法继续跑。
  • I have a blister on my heel because my shoe is too tight.鞋子太紧了,我脚后跟起了个泡。
5 blisters 8df7f04e28aff1a621b60569ee816a0f     
n.水疱( blister的名词复数 );水肿;气泡
参考例句:
  • My new shoes have made blisters on my heels. 我的新鞋把我的脚跟磨起泡了。 来自《现代汉英综合大词典》
  • His new shoes raised blisters on his feet. 他的新鞋把他的脚磨起了水疱。 来自《简明英汉词典》
6 opt a4Szv     
vi.选择,决定做某事
参考例句:
  • They opt for more holiday instead of more pay.他们选择了延长假期而不是增加工资。
  • Will individual schools be given the right to opt out of the local school authority?各个学校可能有权选择退出地方教育局吗?
7 opted 9ec34da056d6601471a0808ebc89b126     
v.选择,挑选( opt的过去式和过去分词 )
参考例句:
  • She was co-opted onto the board. 她获增选为董事会成员。
  • After graduating she opted for a career in music. 毕业后她选择了从事音乐工作。
8 compassion 3q2zZ     
n.同情,怜悯
参考例句:
  • He could not help having compassion for the poor creature.他情不自禁地怜悯起那个可怜的人来。
  • Her heart was filled with compassion for the motherless children.她对于没有母亲的孩子们充满了怜悯心。
9 genetically Lgixo     
adv.遗传上
参考例句:
  • All the bees in the colony are genetically related. 同一群体的蜜蜂都有亲缘关系。
  • Genetically modified foods have already arrived on American dinner tables. 经基因改造加工过的食物已端上了美国人的餐桌。 来自英汉非文学 - 生命科学 - 基因与食物
10 genes 01914f8eac35d7e14afa065217edd8c0     
n.基因( gene的名词复数 )
参考例句:
  • You have good genes from your parents, so you should live a long time. 你从父母那儿获得优良的基因,所以能够活得很长。 来自《简明英汉词典》
  • Differences will help to reveal the functions of the genes. 它们间的差异将会帮助我们揭开基因多种功能。 来自英汉非文学 - 生命科学 - 生物技术的世纪
11 defective qnLzZ     
adj.有毛病的,有问题的,有瑕疵的
参考例句:
  • The firm had received bad publicity over a defective product. 该公司因为一件次品而受到媒体攻击。
  • If the goods prove defective, the customer has the right to compensation. 如果货品证明有缺陷, 顾客有权索赔。
12 disorders 6e49dcafe3638183c823d3aa5b12b010     
n.混乱( disorder的名词复数 );凌乱;骚乱;(身心、机能)失调
参考例句:
  • Reports of anorexia and other eating disorders are on the increase. 据报告,厌食症和其他饮食方面的功能紊乱发生率正在不断增长。 来自《简明英汉词典》
  • The announcement led to violent civil disorders. 这项宣布引起剧烈的骚乱。 来自《简明英汉词典》
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