第2827期:Huntington's disease successfully treated for first time

Huntington's is one of the most brutal and devastating of all diseases. It's caused by a faulty gene which progressively destroys brain cells, which then affects all parts of the body. Symptoms tend to first appear in your 30s or 40s, leading to death within 20 years.亨廷顿病是最凶残和最具破坏性的疾病之一。这种疾病是由一种基因缺陷引起的，这种基因缺陷会渐进性地破坏脑细胞，进而影响身体的所有部位。亨廷顿病的症状通常在 30 多岁或 40 多岁时首次出现，然后会在 20 年内导致死亡。The treatment is a gene therapy which is injected deep into the brain during lengthy surgery. The drug enters cells and delivers a new piece of DNA, which blocks the production of a faulty protein. The trial involved 29 patients. Only the topline results have been released by the company, but these show that three years after surgery, the disease was slowed by an average of 75%, based on measures including cognition, movement and the ability to manage in daily life.本次用于治疗这种疾病的新手段是一种基因疗法，这种疗法通过漫长的手术将药物注射到大脑深处。药物会进入细胞并传递一段新的 DNA，从而阻断有缺陷的蛋白质的产生。这个疗法的试验有 29 名病人参与。相关公司只公布了试验的主要结果，但这些结果显示在手术三年后，疾病的发展速度被平均减缓了 75%，这一结论基于包括认知能力、运动能力和应对日常生活能力等多项指标。Researchers at University College London, who were part of the trial, said the results were spectacular and would give hope to thousands of patients affected by the disease, and to their children, who have a 50/50 chance of inheriting the condition.参与了本次试验的伦敦大学学院的研究人员表示，试验结果令人惊叹，将能为成千上万名罹患这一疾病的患者带来希望，对他们有 50% 的概率会经遗传获得该疾病的子女来说也是如此。The drug could be licensed as soon as next year. It is certain to be expensive. Another gene therapy costs more than £2 million per patient.该药物最早在明年就可能获得上市许可。它肯定会十分昂贵。另一种基因疗法治疗每个病人的费用超过了 200 万英镑。

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