美国国家公共电台 NPR Gene Therapy Shows Promise For A Growing List Of Diseases

 

STEVE INSKEEP, HOST:

Now we have a story about the power of science to heal. For decades scientists have been dreaming about curing diseases with gene¹ therapy. Those dreams have not become reality, but there's growing excitement now. NPR health correspondent Rob Stein reports.

ROB STEIN, BYLINE²: Natalie Wheatley was terrified there might be something wrong with her baby even before he was born. He barely moved during her pregnancy³, and after he was born, doctors told her her worst fears were true. He had a terrible incurable⁴ genetic⁵ disease that was destroying his muscles.

NATALIE WHEATLEY: Basically told me he wouldn't make it to his first birthday, take him home and love him and spend as much time with him as you could. And I'm going to be honest with you. I was devastated⁶, devastated.

STEIN: When Christian⁷ Guardino was born, he was also diagnosed with a rare genetic disease. His wasn't fatal but was destroying his vision.

CHRISTIAN GUARDINO: I wouldn't be able to walk around outside by my own. It would be incredibly dark, blurry⁸ and sort of watching your world fade away.

STEIN: But Wheatley's son, Eli, is still alive. He's now 3 and seems to be thriving in Lebanon, Ky.

WHEATLEY: He just started preschool in September. He goes to preschool alone. He eats in the cafeteria with all the other kids. He's doing extremely well, extremely well. It's been amazing. Truly amazing.

STEIN: And Christian Guardino, who is 17 and lives in Patchogue, N.Y., can now see things he only dreamed about.

GUARDINO: I was able to see things for the first time, like the moon. I was able to see stars for the first time, fireworks and all these amazing things I've never been able to see before.

STEIN: Eli Wheatley and Christian Guardino are among a growing number of patients whose lives are being saved or radically⁹ changed by gene therapy.

DAVID WILLIAMS: This is really an important time in gene therapy.

STEIN: That's David Williams. He's a Harvard scientist who recently reported that gene therapy could cure kids suffering from yet another awful genetic disorder¹⁰, the fatal brain disease made famous by the movie "Lorenzo's Oil."

WILLIAMS: It really shows us that we're able to harness this therapy for some pretty terrible diseases so that's just great.

STEIN: Scientists thought this would happen a long time ago, but the first attempts to save people with defective¹¹ genes¹² by giving them new healthy genes fizzled. And Carrie Wolinetz of the National Institutes of Health says some volunteers in early experiments ended up getting cancer. Some even died.

CARRIE WOLINETZ: And that caused a setback¹³ in the field which caused a lot of concern that maybe gene therapy was not ready for prime time.

STEIN: And may never be. But scientists went back to their drawing boards to come up with better, safer ways to deliver healthy genes into people's bodies, and that finally seems to be paying off.

WOLINETZ: I think we have reached a point of maturation in the science and in some of the new approaches to gene therapy that have allowed us to make rapid advancements¹⁴ in a fairly short period of time. So it's a very exciting time for the field.

STEIN: Now, gene therapy has still only been tested on a small number of patients for relatively¹⁵ short periods of time. A lot more patients will have to be studied for a lot longer before anyone really knows how well it works, for how long and whether it's really safe. Another big question is the price tag. These new gene therapies cost hundreds of thousands of dollars, perhaps even a million dollars for each patient. And Peter Bach of Memorial Sloan Kettering says that's just too much.

PETER BACH: Gene therapies, we've been hearing for years that they were going to be a million dollars before we had any product. So now when they come out, everyone's like, well, we expect them to be a million dollars, simply because it's been repeated so much.

STEIN: Whatever the questions and cost, people who have been helped by gene therapy are just thrilled. Here's Christian Guardino again, the teenager who regained¹⁶ his vision.

GUARDINO: I think that the gene therapy is a miracle, and I think I can't imagine my life without gene therapy. I can't imagine what my life would be like without it.

STEIN: Natalie Wheatley, whose son, Eli, got gene therapy says he keeps getting better.

WHEATLEY: I see progress every day. I see progress every day so that to me offers hope that, you know, gene therapy has saved his life. And I think eventually gene therapy will give the world hope. That's my hope, anyways.

STEIN: The Food and Drug Administration could approve the first gene therapy for a genetic disorder soon, the one Christian got to save his vision. Meanwhile scientists are starting to test gene therapies for a long list of diseases including many that are much more common.

Rob Stein, NPR News.

1 gene   

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。

2 byline   

n.署名；v.署名

参考例句：

• His byline was absent as well.他的署名也不见了。
• We wish to thank the author of this article which carries no byline.我们要感谢这篇文章的那位没有署名的作者。

3 pregnancy   

n.怀孕，怀孕期

参考例句：

• Early pregnancy is often accompanied by nausea.怀孕早期常有恶心的现象。
• Smoking during pregnancy increases the risk of miscarriage.怀孕期吸烟会增加流产的危险。

4 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。

5 genetic   

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。

6 devastated   

v.彻底破坏( devastate的过去式和过去分词)；摧毁；毁灭；在感情上（精神上、财务上等）压垮adj.毁坏的；极为震惊的

参考例句：

• The bomb devastated much of the old part of the city. 这颗炸弹炸毁了旧城的一大片地方。
• His family is absolutely devastated. 他的一家感到极为震惊。

7 Christian   

adj.基督教徒的；n.基督教徒

参考例句：

• They always addressed each other by their Christian name.他们总是以教名互相称呼。
• His mother is a sincere Christian.他母亲是个虔诚的基督教徒。

8 blurry   

adj.模糊的；污脏的，污斑的

参考例句：

• My blurry vision makes it hard to drive. 我的视力有点模糊，使得开起车来相当吃力。 来自《简明英汉词典》
• The lines are pretty blurry at this point. 界线在这个时候是很模糊的。 来自《简明英汉词典》

9 radically   

ad.根本地，本质地

参考例句：

• I think we may have to rethink our policies fairly radically. 我认为我们可能要对我们的政策进行根本的反思。
• The health service must be radically reformed. 公共医疗卫生服务必须进行彻底改革。

10 disorder   

n.紊乱，混乱；骚动，骚乱；疾病，失调

参考例句：

• When returning back,he discovered the room to be in disorder.回家后，他发现屋子里乱七八糟。
• It contained a vast number of letters in great disorder.里面七零八落地装着许多信件。

11 defective   

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 genes   

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

13 setback   

n.退步，挫折，挫败

参考例句：

• Since that time there has never been any setback in his career.从那时起他在事业上一直没有遇到周折。
• She views every minor setback as a disaster.她把每个较小的挫折都看成重大灾难。

14 advancements   

n.（级别的）晋升( advancement的名词复数 )；前进；进展；促进

参考例句：

• Today, the pace of life is increasing with technological advancements. 当今， 随着科技进步，生活节奏不断增快。 来自《现代汉英综合大词典》
• Great advancements in drought prediction have been made in recent years. 近年来，人们对干旱灾害的预报研究取得了长足的进步。 来自互联网

15 relatively   

adv.比较...地，相对地

参考例句：

• The rabbit is a relatively recent introduction in Australia.兔子是相对较新引入澳大利亚的物种。
• The operation was relatively painless.手术相对来说不痛。

16 regained   

复得( regain的过去式和过去分词 )； 赢回； 重回； 复至某地

参考例句：

• The majority of the people in the world have regained their liberty. 世界上大多数人已重获自由。
• She hesitated briefly but quickly regained her poise. 她犹豫片刻，但很快恢复了镇静。