英語閱讀 學英語,練聽力,上聽力課堂! 注冊 登錄
> 輕松閱讀 > 雙語閱讀 >  內容

將DNA植入眼睛治療視力喪失的療法有望在美獲批

所屬教程:雙語閱讀

瀏覽:

2017年11月19日

手機版
掃描二維碼方便學習和分享
A pioneering treatment that can reverse a form of childhood vision loss by inserting DNA directly into the eye is on track for regulatory approval in the US, after a panel of scientists endorsed unanimously the “gene therapy” being developed by Spark Therapeutics.

一種將DNA直接植入眼睛以逆轉某類兒童視力喪失的開創(chuàng)性療法,有望得到美國監(jiān)管機構的批準。此前,一個由科學家組成的顧問委員會對這種由Spark Therapeutics研發(fā)的“基因療法”表示了一致的支持。

If the US Food and Drug Administration accepts the recommendation of its advisory committee, as it tends to, it would mark the agency’s first approval of such a treatment and herald a new era of medicine where diseases are tackled by inserting functioning copies of genes that are missing or mutated.

如果美國食品藥品監(jiān)督管理局(FDA)接受其顧問委員會的建議(往往會接受),將標志著該機構首次批準此類療法,并預示著一個新藥物時代的到來,即通過植入缺失或變異基因的運轉正常的副本來治療疾病。

Gene therapies seek to tackle illness at its biological source, opening up the possibility of drugs that can be delivered just once rather than on a regular basis. In a reference to the potentially curative properties of such treatments, Spark’s shares are listed on Nasdaq under the ticker ONCE.

基因療法尋求從疾病的生物源頭治療疾病,這使得生產出只需投用一次(而不是要定期投用)的藥物成為可能。Spark在納斯達克(Nasdaq)上市的股票的代碼是ONCE,指代這種療法可能藥到病除的特性。

Such therapy also opens up the possibility of finding better treatment options for a string of genetically-driven illnesses that have been hard to tackle with traditional medicine, such as cystic fibrosis and Huntington’s disease.

這種療法還使得為一系列由基因導致的疾病找到更好的治療選擇成為可能,這類疾病很難依靠傳統(tǒng)藥物治療,例如囊性纖維化和亨廷頓氏病(Huntington's disease)。

While representing a huge scientific advance, such one-off treatments are expected to be hugely expensive and threaten to put a strain on already cash-strapped healthcare systems that are structured to pay for chronic therapies.

盡管這代表著巨大的科學進步,但這種一次性療法的價格預計將極其高昂,可能會給本已資金緊張的醫(yī)療系統(tǒng)帶來壓力,這些醫(yī)療系統(tǒng)的設計初衷是為慢性療法買單。

Philadelphia-based Spark will not announce a price until after the drug has been approved but analysts and investors expect it to be in excess of $1m, which would make it one of the most expensive therapies of all time.

總部位于費城的Spark要等到藥物獲批后才會宣布價格,但分析師和投資者預計,其價格將超過100萬美元,令其成為有史以來最昂貴的療法之一。

The therapy, known as Luxturna, is being developed for a subset of patients with a rare inherited disorder known as Leber congenital amaurosis, who number about 6,000 in the US, Europe and other markets that Spark hopes to enter. People with LCA suffer severe vision loss and are at high risk of going completely blind.

這種名為Luxturna的療法,是為部分患有名為“萊伯氏先天性黑蒙”(LCA)的罕見遺傳性疾病的患者研發(fā)的。在美國、歐洲和其他市場,這種患者的數(shù)量約為6000人,Spark希望進入這塊市場?;加羞@種疾病的人視力嚴重喪失,有很高風險變得徹底失明。
 


用戶搜索

瘋狂英語 英語語法 新概念英語 走遍美國 四級聽力 英語音標 英語入門 發(fā)音 美語 四級 新東方 七年級 賴世雄 zero是什么意思大慶市龍鳳廠前教師樓英語學習交流群

網(wǎng)站推薦

英語翻譯英語應急口語8000句聽歌學英語英語學習方法

  • 頻道推薦
  • |
  • 全站推薦
  • 推薦下載
  • 網(wǎng)站推薦