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- PTC124 is an orally administered small molecule that targets a type of disease-causing genetic error known as a nonsense mutation, or premature stop codon. PTC124是一种口服式小分子化合物,它的标的是针对一些因为”无义”基因突变的疾病。
- It would not be suitable for treating different mutations in the dystrophin gene, or diseases not caused by nonsense mutations. 它对肌营养不良基因的其他类型的变异或非无意突变导致的疾病无效。
- PTC124 works by binding to a part of the cell called the ribosome, which translates genetic code into protein, and allows it to ignore nonsense mutations. PTC124通过结合细胞中的核糖体而发挥作用,核糖体负责将基因密码翻译为蛋白,而PTC124容许它忽略无意义突变。
- The company’s current trial of PTC124 in DMD will analyze results from 165 boys with the disease and known nonsense mutations who are at least 5 years old and are still walking. 公司目前关于PTC124治疗DMD的实验将分析165个携带有无义突变的的男孩,他们至少应该五岁以上并且可以行走。
- Furthermore,nonsense mutations(e.g.Y142X and C679X)or new missense variants(e.g.R46L)have recently been found to be associated with a reduction in LDL cholesterol and to reduce the incidence of coronary heart disease(CHD). 无义突变Y142X、C679X及错义变异R46L均可降低LDL胆固醇和冠心病发病率。
- Nobody believes such nonsense in this day and age. 如今没人相信这种无稽之谈。
- I've never heard such nonsense in all my born days! 我一生中从未听到过这种胡言乱语。
- What the squire said is absolutely nonsense! 这个乡绅的话纯属一派胡言!
- Congenital afibrinogenemia associated with a novel nonsense mutation in the FGA gene 一种新的FGA基因无义突变导致遗传性无纤维蛋白原血症
- Our teacher has mutation of sounds suddenly. 我们的老师突然语音发生了变化。
- Nonsense, you must have misheard. 瞎说,你准是听错了。
- It was sheer nonsense to trust them. 相信他们纯粹是胡闹。
- Oh, go to hell! Don't talk such nonsense! 去你的吧!别这么胡说八道!
- He won't stand any nonsense from the staff. 他决不容忍员工胡作非为。
- Stop that nonsense, children, and get into bed! 别胡闹了,孩子们,都睡觉去!
- Keywords nonsense mutation;genetic disease;Duchenne muscular dystrophy(DMD);cystic fibrosis;PTC124; 无义突变;遗传性疾病;杜兴肌营养不良;囊性纤维病变;PTC124;
- The fool is talking stark nonsense. 这傻瓜完全是在胡扯。
- He's been talking dashed nonsense! 他简直是一派胡言!
- Don't let him fool you with nonsense like that. 别让他用那种胡说八道来愚弄你。
- She is forever chattering nonsense. 她老是喋喋不休地胡说八道。