基因沉寂藥物有望改善卟啉病患者生活

一種利用基因沉寂技術(shù)的創(chuàng)新型藥物有望被納入英國國民醫(yī)療體系（NHS），用來治療那些生活在極度病痛中的人。該藥物可以治療急性間歇性卟啉病，這種遺傳性疾病可導(dǎo)致人們無法工作或正常生活。

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

卟啉病可造成強(qiáng)烈的疼痛，以至于患者每天都要服用強(qiáng)效的類鴉片止痛藥。嚴(yán)重發(fā)作時(shí)甚至需要入院治療。

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

基因沉寂技術(shù)不是要控制病痛，而是從這種遺傳病的根本病因著手治療，并對抗有毒蛋白質(zhì)在體內(nèi)的蓄積。

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%.

該藥物不會改變病人的 DNA（脫氧核糖核酸），但會抑制部分 DNA，從而阻止這類蛋白質(zhì)的產(chǎn)生。試驗(yàn)表明，它幫助減少了75%的病癥發(fā)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.

為英格蘭地區(qū)提供藥物咨詢的國家衛(wèi)生與保健優(yōu)化研究所表示，這種治療方法將改善人們的生活質(zhì)量，且性價(jià)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.

卟啉病是一種罕見的疾病。但這種新療法顯示了基因沉寂療法是如何開始改變醫(yī)學(xué)的?？茖W(xué)家正在研究如何將這種技術(shù)應(yīng)用于治療從阿茲海默癥到高血壓等各類疾病。