????DNA療法

2003年,，一支為美國能源部（Department of Energy）和國立衛(wèi)生研究院（National Institutes of Health）工作的研究團(tuán)隊(duì)完成了一項(xiàng)長達(dá)13年之久的研究項(xiàng)目,。這個(gè)項(xiàng)目名叫人類基因組計(jì)劃。計(jì)劃的目標(biāo)之一是確定并繪制人類DNA每一個(gè)基因的圖譜,。目前科學(xué)界正在研究這些數(shù)據(jù),，并且猜測：“下一步會(huì)是什么？”

????其中一個(gè)答案是靶向療法,，也就是并不僅僅以減輕癥狀為目的,，同時(shí)還針對(duì)患者的基因構(gòu)成進(jìn)行治療。

????過去幾年里,，靶向療法領(lǐng)域已經(jīng)出現(xiàn)了一些突破,。2011年，賓西法尼亞大學(xué)（University of Pennsylvania）的一群科學(xué)家通過調(diào)動(dòng)病人自身的白血球,，有效地幫助白血病患者戰(zhàn)勝了疾病,。同樣是在2011年，英國的研究人員通過向B型血友病患者注入改良過的基因,，在治療這種基因病上取得了令人滿意的效果,。2012年，科學(xué)界還在該通過靶向療法，在治療遺傳性失明上取得了更加重大的進(jìn)展,。

????沃頓商學(xué)院麥克科技創(chuàng)新中心另一位常務(wù)董事喬治?戴伊指出：“靶向療法意味著整個(gè)醫(yī)療行業(yè)的轉(zhuǎn)型,。”他說：“大型制藥公司總是以藥品為中心,，”也就是說患者每天都需要服藥,，而“基因療法則是一勞永逸?！?/p>

????“我想在20年后,，對(duì)基因療法的預(yù)測將開始變稱現(xiàn)實(shí)?！?/p>

????DNA Rx

????In 2003, a team of researchers working for the Department of Energy and the National Institutes of Health completed a 13-year long effort called the Human Genome Project. The goal of the project was, among other things, to identify and map every gene in human DNA. For a while, the scientific community has been looking at the data and wondering, "So, what's next?"

????One answer is targeted therapy, medical treatments that do not only address symptoms, but also respond to a patient's genetic makeup.

????The past couple of years have yielded breakthroughs in this field. In 2011, a team of scientists at the University of Pennsylvania effectively treated leukemia patients by engineering their own white blood cells to fight the disease. Also in 2011, researchers in Britain achieved promising results in treating the genetic disease hemophilia B by injecting patients with a re-engineered version of the gene. 2012 brought even more advancements, using targeted therapy to treat inherited blindness.

????"Talk about transforming an industry," says George Day, co-director of the Mack Center at Wharton. "Big Pharma has always been pill-based," he says, meaning patients need daily dosage, whereas "gene therapy is one and done.

????"I think finally after 20 years, the promise of gene therapy is starting to be realized."