Gene Therapy to Treat Hemophilia

（单词翻译）

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