| 查看: 551 | 回复: 4 | |||
| 当前主题已经存档。 | |||
[交流]
非常好的背诵材料，来自美国科学杂志的
|
|||
|
Gene Therapy Helps Blind Children See A single injection of DNA into the eyes of four children born with a blindness-causing disease has given them enough vision to walk without help. The study, published today, confirms that if patients with this disease are given gene therapy early in life, the results can be dramatic. Several clinical trials in the United States and Europe have been using gene therapy to treat a disease called Leber's congenital amaurosis (LCA), which affects about 3000 people in the United States. Those born with LCA start losing their sight at birth and are completely blind by age 40. Children born with one form, LCA2, have defects in a gene called RPE65 that helps the retina's light-sensing cells make rhodopsin, a pigment needed to absorb light. Without rhodopsin, the photoreceptor cells gradually die. In 2001, researchers at the University of Pennsylvania (Penn) showed that they could partially restore sight to blind dogs with this defect by injecting a good copy of RPE65 into their eyes. Two years ago, the Penn team began a small safety study of the therapy in humans with collaborators at the Children's Hospital of Philadelphia. They injected each patient's worse eye with a modified virus carrying the RPE65 gene. Early results from this trial and a similar study in the United Kingdom published in April 2008 showed that four of six young adults with LCA2 who received the treatment could later sense more light and perform better in an obstacle course. But the Penn researchers knew from their studies in animals that children should improve even more because they have more intact retinal tissue than adults do. Today in an online paper in The Lancet, their team and collaborators in Europe report full study results for three of the adults they treated earlier and nine more patients, including four children ages 8 to 11. The children gained more light sensitivity than the adults did--their light sensitivity increased as much as four orders of magnitude, versus one--and they made far fewer mistakes in an obstacle course. One patient, Corey Haas, appears in a video 3 months after the treatment breezing through the obstacle course, following arrows and avoiding objects that he cannot see with his treated eye covered. Corey, 9, told reporters at a press conference this week that for the first time he can recognize faces, play baseball, read large print books, and ride his bike around his neighborhood alone. "It has helped him tremendously," said his father, Ethan Haas. Gene Therapy Helps Blind Children See A single injection of DNA into the eyes of four children born with a blindness-causing disease has given them enough vision to walk without help. The study, published today, confirms that if patients with this disease are given gene therapy early in life, the results can be dramatic. Several clinical trials in the United States and Europe have been using gene therapy to treat a disease called Leber's congenital amaurosis (LCA), which affects about 3000 people in the United States. Those born with LCA start losing their sight at birth and are completely blind by age 40. Children born with one form, LCA2, have defects in a gene called RPE65 that helps the retina's light-sensing cells make rhodopsin, a pigment needed to absorb light. Without rhodopsin, the photoreceptor cells gradually die. In 2001, researchers at the University of Pennsylvania (Penn) showed that they could partially restore sight to blind dogs with this defect by injecting a good copy of RPE65 into their eyes. Two years ago, the Penn team began a small safety study of the therapy in humans with collaborators at the Children's Hospital of Philadelphia. They injected each patient's worse eye with a modified virus carrying the RPE65 gene. Early results from this trial and a similar study in the United Kingdom published in April 2008 showed that four of six young adults with LCA2 who received the treatment could later sense more light and perform better in an obstacle course. But the Penn researchers knew from their studies in animals that children should improve even more because they have more intact retinal tissue than adults do. Today in an online paper in The Lancet, their team and collaborators in Europe report full study results for three of the adults they treated earlier and nine more patients, including four children ages 8 to 11. The children gained more light sensitivity than the adults did--their light sensitivity increased as much as four orders of magnitude, versus one--and they made far fewer mistakes in an obstacle course. One patient, Corey Haas, appears in a video 3 months after the treatment breezing through the obstacle course, following arrows and avoiding objects that he cannot see with his treated eye covered. Corey, 9, told reporters at a press conference this week that for the first time he can recognize faces, play baseball, read large print books, and ride his bike around his neighborhood alone. "It has helped him tremendously," said his father, Ethan Haas. These videos show Corey Haas walking through an obstacle course 3 months after receiving a gene therapy injection in one eye. In the first video, his treated eye is patched. In the second video made 10 minutes later, the course has been rearranged and his untreated eye is patched. The LCA2 trials are a rare success for the field of gene therapy, which has also cured children with the immune disorder known as bubble boy disease. And they should pave the way for treating more vision disorders. "It's an incredible launching pad to be able to target other diseases," says Penn gene therapy researcher Jean Bennett, who led the study. |
回复此楼» 猜你喜欢
上海大学实验技术岗位非升即走
已经有9人回复
-
云南大学材料与能源学院解琳课题组钙钛矿博士招生
已经有4人回复
-
博士申请
已经有6人回复
-
评审有感
已经有11人回复
-
面上本子正文33页，违规吗？会被低分嘛？
已经有10人回复
-
国自然上会要求
已经有9人回复
-
今年审到国自然15份，谈谈感受
已经有16人回复
-
考博自荐
已经有6人回复
-
青C资助名额大幅增加！
已经有16人回复
-
重磅！青年科学基金项目（C类）资助增幅预计超过50%
已经有10人回复
» 本主题相关商家推荐: (我也要在这里推广)
2楼2009-10-25 20:02:00
 |
3楼2009-10-28 19:31:33
4楼2009-10-28 22:04:13
当然背啊，不过即使你不愿意背，读熟也很好啊，这些都是最新的文章。非常好的5楼2009-10-29 09:29:57
30