身体发肤可治病

身体发肤可治病 科学家们最近在一种罕见遗传疾病的治疗上取得了很大的进展。他们的方法是从患者身上提取很少量的皮肤薄片和头发，再借此制造出健康的细胞。 这项研究第一次证实了可以通过修复人类细胞中的遗传缺陷，再以修复后健康的细胞替代有缺陷的细胞来治疗疾病。 在Nature杂志的一篇报导中，研究者描述了他们如何从6个患有范康尼贫血症-一种罕见的遗传血液疾病-的患者身上提取皮肤和头发细胞。范康尼贫血症是由遗传缺陷引发的疾病，会导致骨髓功能衰竭，甚至更容易罹患癌症，例如血癌。先天性的范康尼患者通常在早期儿童期就可以被诊断，但是很少能活过3岁。 整个过程分为三个步骤:从患者身上提取细胞之后，首先，研究者使用基因疗法来修复细胞中有缺陷的DNA，然后，他们用一种叫做“细胞重新编程”的技术将这些细胞转为健康的干细胞--唯一可以在人体任何一种组织中生长的细胞。 最后，他们在陪替式培养皿中培养这些干细胞，让它们长成早期骨髓细胞，原则上这样就可以植入患者体内来治病了。 “我们还没有治愈患病的人，可是我们已经治愈患病的细胞了”加州La Jolla市萨克生物研究学院负责此项研究的Juan-Carlos Izpisúa Belmonte说“(但是)理论上我们是可以把它植入人体，并治愈这种疾病的。” 目前在全球有很多干细胞科学家在研究类似技术以治疗其他遗传疾病，而这一研究结果将会提升所有这些科学家的信心。这项技术之所以这么受关注，是因为它使用的是患者自己的细胞，不会引起免疫系统的排异反应。 加州的这个团队并没有把健康细胞注入那6个患者体内，因为研究过程中使用的无害病毒可能会把细胞转为肿瘤。科学家们正在寻找替代方案，希望可以不再依赖这些病毒。 伦敦国王学院的分子遗传学教授Chris Matthews说:“将来有可能实现将修正的干细胞转回患者体内这一目标，但是从实验室到临床，还有很多工作要做。”伦敦大学学院的再生医学教授Chris Mason补充说:“如今对于这种疾病根本没有什么治疗方法，更别说治愈了。然而这项研究结果毫无疑问将是第一次为应对这种困境提供了希望。” Hair and skin cells could cure diseases, say scientists Scientists have taken a big step towards treating a rare inherited disease by creating healthy cells from flakes of skin and strands of hair plucked from patients. The study is the first to demonstrate that it is possible to repair genetic faults in human cells and make batches of healthy replacements that could be used to treat a disease. In a report in the journal Nature, researchers describe how they took skin and hair cells from six patients with a rare inherited blood disorder called Fanconi anaemia. The disease is caused by a genetic defect that leads to bone marrow failure and a greater risk of cancers, such as leukaemia. People who are born with Fanconi anaemia are usually diagnosed in early childhood and rarely survive beyond 30-years-old. In a three-stage procedure, the researchers used gene therapy to fix the faulty DNA in the cells they had taken from patients. Next, they used a technique called cell reprogramming to convert these cells into healthy stem cells, which are unique in being able to grow into any kind of tissue in the body. In the final stage of the process, the researchers grew the stem cells in petri dishes into early stage bone marrow cells, which in principle could be injected into patients to treat their condition. "We haven't cured a human being, but we have cured a cell," said Juan-Carlos Izpisúa Belmonte, who led the study at the Salk Institute for Biological Studies in La Jolla, California. "[But] in theory we could transplant it into a human and cure the disease." The feat will raise the hopes of stem cell scientists all over the world who are working on similar techniques to treat other inherited diseases. The technique is appealing because it uses a patient's own cells, which would not be rejected by the immune system. The California team did not inject the healthy cells back into the six patients because harmless viruses used in the procedure could cause the cells to turn into tumours. Scientists are developing alternatives that do not rely on viruses. Chris Matthews, professor of molecular genetics at King's College London, said: "In future it may become possible to transfer the corrected stem cells back into the patient, but much work remains to be done before this can be transferred from the lab bench to the bedside."Chris Mason, professor off regenerative medicine at University College London, added: "There is no doubt that this paper will be the first of many to offer hope for conditions where today there is no real therapy, let alone a cure."