基因沉寂药物有望改善卟啉病患者生活

一种利用基因沉寂技术的创新型药物有望被纳入英国国民医疗体系（NHS），用来治疗那些生活在极度病痛中的人。该药物可以治疗急性间歇性卟啉病，这种遗传性疾病可导致人们无法工作或正常生活。

Porphyria can be so painful that people take potentopioid painkillersevery day. Severe attacks need hospital treatment.

卟啉病可造成强烈的疼痛，以至于患者每天都要服用强效的类鸦片止痛药。严重发作时甚至需要入院治疗。

Rather than managing the pain, genesilencing gets to the root cause of the geneticdisease and the build-up of toxicproteins in the body.

基因沉寂技术不是要控制病痛，而是从这种遗传病的根本病因着手治疗，并对抗有毒蛋白质在体内的蓄积。

The drug does not alter people's DNAbut mutes parts of it to block that protein production. Trials showed it helped cut attacks by 75%.

该药物不会改变病人的 DNA（脱氧核糖核酸），但会抑制部分 DNA，从而阻止这类蛋白质的产生。试验表明，它帮助减少了75%的病症发作。

The National Institute for Health and Care Excellence⁷, which advises on drugs in England, said the therapy would improve people's quality of life and was value for money.

为英格兰地区提供药物咨询的国家卫生与保健优化研究所表示，这种治疗方法将改善人们的生活质量，且性价比高。

Porphyria is a rare disease. But this new treatment shows how gene silencing therapies are starting to change medicine. Scientists are investigating how the technology can be used in conditions from Alzheimer's to high blood pressure.

卟啉病是一种罕见的疾病。但这种新疗法显示了基因沉寂疗法是如何开始改变医学的。科学家正在研究如何将这种技术应用于治疗从阿兹海默症到高血压等各类疾病。