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| ScienceDaily (July 13, 2010) — Italian scientists pioneering a new gene transfer treatment for the blood disorder β-thalassemia have successfully completed preclinical trials, claiming they can correct the lack of beta-globin (ß-globin) in patients' blood cells which causes the disease. The research, published in EMBO Molecular Medicine, reveals how gene therapy may represent a safe alternative to current cures that are limited to a minority of patients. 《每日科学》2010年7月13日报道 —— 率先为β地中海贫血症患者开展新的基因转移疗法的意大利科学家已成功完成临床前试验，宣告他们有能力纠正患者血细胞缺乏ß-球蛋白问题（正是这种缺乏导致ß地贫）。目前治疗ß地贫的疗法仅局限于少数患者，而这项发表于《EMBO分子医学》（EMBO为欧洲分子生物学组织的英文缩写——译者）的研究则揭示基因疗法有可能成为目前疗法的安全的替代方法。 The disorder β-thalassemia, also known as Cooley's anemia, is caused when a patient cannot produce enough of the ß-globin component of haemoglobin, the protein used by red blood cells to carry oxygen around the body. The lack of ß-globin causes life threatening anemia, leading to severe damage of the body's major organs. The condition is most commonly found in Mediterranean, Middle Eastern and Asian populations. β地中海贫血症，或库利氏贫血，在患者无法产生血红蛋白中的ß-球蛋白成分时就会出现。ß-球蛋白是红血球用以携带体内所需要的氧的蛋白质。ß-球蛋白的缺乏可致贫血，损坏主要器官而危及生命。这一症状在地中海、中东与亚洲人口中出现得最为普遍。 "Currently treatments are limited to lifelong regular blood transfusions, and iron chelation to prevent fatal iron overload. The alternative is bone marrow transplantation, an option open to less than 25% of patients," said Dr Giuliana Ferrari from the San Raffaele Telethon Institute for Gene Therapy in Milan. "Our research has focused on gene therapy: by transplanting genetically corrected stem cells we can restore haemoglobin production and overcome the disorder." “目前的治疗局限于终身定期输血与铁螯合剂以防止致命的铁质过载。相关替代方法是骨髓移植，但只有少于25%的患者能够作这种选择。我们的研究关注于基因疗法；通过移植经过基因工程纠正的干细胞，从而恢复血红蛋白生产并克服这一症状。” 位于意大利米兰的San Raffaele Telethon 基因疗法研究所的Giuliana Ferrari说。 Diseases of the blood are good targets for gene therapy because it is possible to harvest stem cells from the patient's bone marrow. The team developed a tool to deliver the correct gene for ß-globin into these harvested cells, a viral vector they called GLOBE. 血液疾病是基因疗法的优秀目标，因为有可能从患者的骨髓中提取干细胞。该研究团队开发了一种称之为GLOBE的病毒载体工具，利用该工具可以让ß-球蛋白所需要的正确的基因进入所提取的细胞中。 The cells can then be genetically modified with GLOBE to restore hemoglobin production before being re-administered back into the patient via intravenous injections. The important focus of this work was not only to show that GLOBE can restore haemoglobin production in human cells, but that this genetic transfer-based approach does not impair the biological features of the cells and is not associated with any intrinsic risk for the human genome. 由于GLOBE的存在，所提取的细胞会在基因水平上得到修正，从而恢复血红蛋白的生产。接着研究者把纠正后细胞通过静脉注射又送回到患者的体内。本研究工作的重要核心不仅是显示GLOBE能够恢复血红蛋白在人类细胞中的生产，而且显示了这种基于基因传移的方法不会损及细胞的生物学特征，也与人类基因组的任何内在风险无关。 This research is not only crucial for developing a cure for one disease, but as Dr David Williams from the Harvard Medical School says, it may advance the entire discipline of gene therapy research 这一研究不仅对开发能彻底治愈一种疾病的疗法至关重要，而且，诚如来自哈佛医学院的David Williams 博士所言，本研究可能推进了整个基因疗法研究领域的进展！ "This work represents the kind of translational studies that are required to move human investigations forward but are often difficult to fund and publish," said Williams. "Considering the inherent difficulties accompanying human research, studies like those reported in EMBO Molecular Medicine are extremely important for moving the field forward." As the Milan based team can now correct the defective production of beta-globin in patients' blood cells the next step will be to place the corrected cells back into the patient, a step which has already proven successful in mice. “这一工作在转译研究方面具有代表性。大家知道，转译研究类的工作，需要推进人类学术研究向前发展、但发现与发表却经常困难重重，” Williams说，“考虑到人类研究所存在的内在困难，发表在《EMBO分子医学》上的这项研究成果对于推进该领域尤显重要。” 因为这支米兰研究小组现在能够纠正患者血细胞中的ß-球蛋白生产缺陷，下一步将是把纠正后的细胞放回患者体内，并且这一步在小鼠实验已经取得了成功。 Successful gene therapies are the results of very long studies and our research represents the most comprehensive pre-clinical analysis ever performed on cells derived from thalassemic patients" concluded Ferrari. "We believe this study paves the way forward for the clinical use of stem cells genetically corrected using the GLOBE vector." Ferrari总结说：“成功的基因疗法是耗时极为漫长的研究的结果，而我们的实验代表着对取自地中海贫血症患者的细胞的最全面的临床前分析。我们相信本研究为利用GLOBE这一载体工具进行基因矫正的干细胞的临床应用辅平了道路。” |
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