2012年五大科技突破

    DNA疗法

2003年，一支为美国能源部（Department of Energy）和国立卫生研究院（National Institutes of Health）工作的研究团队完成了一项长达13年之久的研究项目。这个项目名叫人类基因组计划。计划的目标之一是确定并绘制人类DNA每一个基因的图谱。目前科学界正在研究这些数据，并且猜测：“下一步会是什么？”

    其中一个答案是靶向疗法，也就是并不仅仅以减轻症状为目的，同时还针对患者的基因构成进行治疗。

    过去几年里，靶向疗法领域已经出现了一些突破。2011年，宾西法尼亚大学（University of Pennsylvania）的一群科学家通过调动病人自身的白血球，有效地帮助白血病患者战胜了疾病。同样是在2011年，英国的研究人员通过向B型血友病患者注入改良过的基因，在治疗这种基因病上取得了令人满意的效果。2012年，科学界还在该通过靶向疗法，在治疗遗传性失明上取得了更加重大的进展。

    沃顿商学院麦克科技创新中心另一位常务董事乔治•戴伊指出：“靶向疗法意味着整个医疗行业的转型。”他说：“大型制药公司总是以药品为中心，”也就是说患者每天都需要服药，而“基因疗法则是一劳永逸。”

    “我想在20年后，对基因疗法的预测将开始变称现实。”

    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."