血友病治疗方法的重大突破
Haemophilia breakthrough
Walking two miles to work used to be unthinkable for Jake Omer.
走两英里路去上班对杰克·奥马尔来说曾是一件难以想象的事情,因为他有先天性A型血友病。
He was born with haemophilia A, a genetic defect that means his blood did not clot.
这是一个基因缺陷,而因此导致血液无法凝结。
The slightest injury used to mean severe bleeding.
即使是最轻微的小伤也会造成大量的流血。
Even a long stroll would cause bleeding in his joints. But no more.
甚至,散步时间稍长一些也会使得他的关节流血。但是现在,这一切都有所改观。
“I think the gene therapy has hopefully given me a new lease of life.
“我觉得基因疗法给了我开启新生活的希望。
It's going to allow me, when as my boys grow up, to be a lot more active with them - to kick footballs around, to climb trees with them, to hopefully run around in the park with them - and not be someone who's got to worry about what I'm doing.”
它使我能够在我的儿子们成长的过程中,更多地跟他们一起玩耍、运动,比如跟他们一起踢球、爬树,或者在公园里奔跑。而不是太需要担心自己的身体状况。”
Jake was one of 13 patients given pioneering gene therapy last year.
杰克是去年接受这项前沿基因治疗的十三位病人中的一位。
A virus was used to give his body new genetic instructions for clotting blood.
他被接种了一种病毒,用来给他的凝血功能下达一种新的遗传指令。
All of the trial patients are off the haemophilia medication and 11 have roughly normal levels of blood clotting proteins.
目前所有参加试验的患者都已停药,其中有十一位患者体内的凝血蛋白差不多达到了正常水平。
“This is huge! It's ground-breaking because the option to think about normalising levels in patients with severe haemophilia is absolutely mind-blowing.
“这是一项巨大的、具有开创性的试验成果,因为它给严重血友病患者的凝血蛋白正常化提供了新的治疗选择。这绝对是个令人称奇的研究成果。
To offer people the potential of a normal life when they have had to inject themselves with Factor VIII every other day to prevent bleeding is transformational.”
相比以前,患者为了能正常生活,每天得给自己注射凝血第八因子来防止出血,这绝对是一次变革。”
Large studies will now take place to see if gene therapy can replace these regular injections and truly transform the lives of patients.
大规模的研究即将开始,这些研究将近一步观察基因治疗是否能取代定期注射,从而使患者的生活彻底改观。