Gene Therapy to Treat Hemophilia

64 基因疗法治疗血友病疗效有限

DATE=6-28-01
TITLE=SCIENCE REPORT - Gene¹ Therapy to Treat Hemophilia
BYLINE=Nancy Steinbach

(Start at 01'02") This is Bill White With the VOA Special English Science Report.
American researchers report (1) limited success using (2) gene therapy to treat the (3) genetic² blood disease (4) hemophilia.
Hemophilia results when a gene fails to produce the (5) protein needed for the blood to (6) clot³, or change from a liquid to a solid. The (7) defective⁴ gene is passed from parents to children.  People with hemophilia suffer uncontrolled bleeding.  This can result in pain, (8) tissue (9) swelling⁵ and (10) permanent damage to (11) joints⁶ and muscles.  
One in every ten-thousand males has the most common kind of hemophilia.  It is extremely (12) rare for  females to have it. Patients can be treated with the missing clotting⁷ substance.  They generally can lead normal lives.
Scientists say gene therapy may be a possible way to (13) cure hemophilia in the future. Most gene therapies use a (14) virus to place a good copy of a gene into a cell that needs it. The new gene helps the body operate normally.  For people with hemophilia, this means that clotting genes⁸ placed in the body would result in blood cells that clot normally.
Researchers consider hemophilia the best disease for gene therapy because it is caused by a single defective gene.  Also, only a small increase in the missing clotting substance could provide good results. Scientists at the Beth (15) Israel Deaconess Medical Center in Boston, Massachusetts carried out the experiment.  They reported the results in the New England Journal of Medicine. They tested gene therapy in six patients with (16) severe hemophilia. 
First, they removed skin cells from the patients' arms.  The researchers grew the cells in the laboratory.  They added copies of the needed gene taken from healthy people.  Then they created hundreds of millions of genetically⁹ changed cells.  They placed these cells into the patients' stomachs.
After four months, the amount of blood clotting substance in the blood increased in four of the six patients.  Some of the patients reported a decrease in bleeding problems.  However, ten months later, the clotting substance was no longer in the patients' blood.  It is not clear if the (17) implanted cells died or the added genes stopped working.
The researchers say the study showed that gene therapy is safe for people with the most common kind of hemophilia.  But others expressed concern about the treatment because the effects were only (18) temporary. 
This VOA Special English Science Report was written by Nancy Steinbach. This is Bill White.

(1) limited [ 5limitid ] adj.有限的, 狭窄的, 缺乏创见的 n.高级快车
(2) gene therapy [ 5WerEpi ] n.基因治疗
(3) genetic [ dVi5netik ] adj.遗传的, 起源的
(4) hemophilia [ 7hi:mE5filiE ] n.血友病
(5) protein [ 5prEuti:n ] n.[生化]蛋白质 adj.蛋白质的
(6) clot [ klCt ] n.(血液等的)凝块 v.(使)凝结
(7) defective [ di5fektiv ] adj.有缺陷的, (智商或行为有)欠缺的 n.有缺陷的人, 不完全变化动词
(8) tissue [ 5tisju: ] n.[生]组织, 连篇
(9) swelling [ 5sweliN ] n.河水猛涨,涨水
(10) permanent [ 5pE:mEnEnt ] adj.永久的, 持久的
(11) joint [ dVCint ] n.接缝, 接合处, 接合点, 关节 adj.共同的, 联合的vt.连接, 接合, 使有接头
(12) rare [ rZE ] adj.稀罕的, 杰出的, 珍贵的, (肉类)半熟的 adv.非常
(13) cure [ kjuE ] v.治愈, 治疗 n.治愈, 痊愈
(14) virus [ 5vaiErEs ] n.[微]病毒, 滤过性微生物, 毒害, 恶毒
(15) Israel [5izreiEl] n.[地名] 以色列
(16) severe [ si5viE ] adj.严厉的, 严格的, 剧烈的, 严重的, 严峻的
(17) implant [ im5plB:nt ] v.灌输
(18) temporary [ 5tempErEri ] adj.暂时的, 临时的, 临时性

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 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。

3 clot   

n.凝块；v.使凝成块

参考例句：

• Platelets are one of the components required to make blood clot.血小板是血液凝固的必须成分之一。
• The patient's blood refused to clot.病人的血液无法凝结。

4 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。

5 swelling   

n.肿胀

参考例句：

• Use ice to reduce the swelling. 用冰敷消肿。
• There is a marked swelling of the lymph nodes. 淋巴结处有明显的肿块。

6 joints   

接头( joint的名词复数 )； 关节； 公共场所（尤指价格低廉的饮食和娱乐场所） （非正式）； 一块烤肉 （英式英语）

参考例句：

• Expansion joints of various kinds are fitted on gas mains. 各种各样的伸缩接头被安装在煤气的总管道上了。
• Expansion joints of various kinds are fitted on steam pipes. 各种各样的伸缩接头被安装在蒸气管道上了。

7 clotting   

v.凝固( clot的现在分词 )；烧结

参考例句：

• The study was also based only on the one anti-clotting drug. 所用抗血栓药物也仅限于一种。 来自互联网
• A plasma protein that is converted into thrombin during blood clotting. 凝血原酶一种血浆蛋白质，在血液凝聚时转化成凝血''。'酵'。''''。'素'。''。 来自互联网

8 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

9 genetically   

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