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Treatment by cell transplant

机译：细胞移植治疗

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"粒细胞一巨噬细胞群刺激因子"(GM-CSF)的受体发生的突变会造成"肺泡蛋白沉积症"(PAP)。这是一种遗传病,会由于表面活性剂的积累而导致呼吸衰竭。这种病目前还没有治疗药物,必须通过肺灌洗将表面活性剂清除。PAP在小鼠模型中可以通过基因矫正的造血干细胞的骨髓移植治愈,但这种方法由于在必要的骨髓清除/免疫抑制过程中的感染此前还没有被证明对人类可行。现在,Bruce Trapnell及同事报告了在将基因矫正的巨噬细胞一次性直接转移到肺中之后小鼠PAP所发生的矫正。基因矫正的巨噬细胞持续存在了至少了一年。自体基因矫正的巨噬细胞的这种肺移植,将会免除对骨髓清除和免疫抑制的需要,对于人类PAP的治疗来说也应当更为可行。%Pulmonary alveolar proteinosis (PAP) is a rare lung disease characterized by the accumulation in the lung of white blood cells called alveolar macrophages that are full of surfactant - a compound of phospholipids and proteins that regulates surface tension in the lung - and of vast amounts of extracellular surfactant. Unravelling the cause of this disease, which was first recognized in 1958,is a story that began in 1994 with the serendipitous discovery that mice lacking the protein GM-CSF, which is important for macrophage maturation and function, had a mysterious lung disease that resembled human PAP. In this issue, Suzuki et al (page 450) add a chapter to this story, reporting that transplanting macrophages that correctly respond to GM-CSF into the lungs of mice lacking the GM-CSF receptor effectively treats their disease.
机译："粒细胞一巨噬细胞群刺激因子"(GM-CSF)的受体发生的突变会造成"肺泡蛋白沉积症"(PAP)。这是一种遗传病,会由于表面活性剂的积累而导致呼吸衰竭。这种病目前还没有治疗药物,必须通过肺灌洗将表面活性剂清除。PAP在小鼠模型中可以通过基因矫正的造血干细胞的骨髓移植治愈,但这种方法由于在必要的骨髓清除/免疫抑制过程中的感染此前还没有被证明对人类可行。现在,Bruce Trapnell及同事报告了在将基因矫正的巨噬细胞一次性直接转移到肺中之后小鼠PAP所发生的矫正。基因矫正的巨噬细胞持续存在了至少了一年。自体基因矫正的巨噬细胞的这种肺移植,将会免除对骨髓清除和免疫抑制的需要,对于人类PAP的治疗来说也应当更为可行。%Pulmonary alveolar proteinosis (PAP) is a rare lung disease characterized by the accumulation in the lung of white blood cells called alveolar macrophages that are full of surfactant - a compound of phospholipids and proteins that regulates surface tension in the lung - and of vast amounts of extracellular surfactant. Unravelling the cause of this disease, which was first recognized in 1958,is a story that began in 1994 with the serendipitous discovery that mice lacking the protein GM-CSF, which is important for macrophage maturation and function, had a mysterious lung disease that resembled human PAP. In this issue, Suzuki et al (page 450) add a chapter to this story, reporting that transplanting macrophages that correctly respond to GM-CSF into the lungs of mice lacking the GM-CSF receptor effectively treats their disease.

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  • 来源
    《Nature》 |2014年第7523期|438-440B1|共4页
  • 作者

    MARY JANE THOMASSEN; MANI S. KAVURU;

  • 作者单位

    Division of Pulmonary, Critical Care and Sleep Medicine, East Carolina University, Greenville, North Carolina 27834, USA;

    Division of Pulmonary and Critical Care Medicine, Thomas Jefferson University and Hospital Philadelphia, Pennsylvania 19107, USA;

  • 收录信息 美国《科学引文索引》(SCI);美国《工程索引》(EI);美国《生物学医学文摘》(MEDLINE);美国《化学文摘》(CA);
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