美国威斯康辛大学教授詹姆斯·汤姆森的科研小组日前成功地在试管内利用万能细胞再现了疾病过程。该小组用重症神经疾病患者的皮肤细胞培养出人工多功能干细胞(iPS细胞)，将这些iPS细胞培育为神经细胞后，在试管内成功再现了神经细胞因疾病死亡的过程。
这是世界首例使用患者iPS细胞重现病症的成功尝试。科研小组在英国科学杂志《自然》上发表了该成果，预计今后可广泛应用于探明病因、研发新药等领域。
科研小组表示“随着时间推移成功观测到了神经细胞出现的异常变化。在患者体内难以观测的病变可以在试管内清晰再现了”。
日本iPS细胞专家、国立成育医疗中心研究所室长阿久津英宪表示，该成果对iPS细胞在治疗疾病、药物研发领域中的现实应用迈进了一大步。曾有专家担心“重现病症会很难”，而该成果体现了研究的飞速发展。用iPS细胞可无限度繁殖病变细胞用于研究，此意义实属非凡。（生物谷Bioon.com）
生物谷推荐原始出处：
Nature advance online publication 21 December 2008 | doi:10.1038/nature07677
Induced pluripotent stem cells from a spinal muscular atrophy patient
Allison D. Ebert1,2, Junying Yu3, Ferrill F. Rose, Jr4, Virginia B. Mattis4, Christian L. Lorson4, James A. Thomson2,3,5 & Clive N. Svendsen1,2,5,6
1 The Waisman Center, and,
2 The Stem Cell and Regenerative Medicine Center, University of Wisconsin-Madison, 1500 Highland Avenue, Madison, Wisconsin 53705, USA
3 The Genome Center and Wisconsin National Primate Research Center, University of Wisconsin-Madison, 425 Henry Mall, Madison, Wisconsin 53706, USA
4 Department of Veterinary Pathobiology, Bond Life Sciences Center, University of Missouri, 1201 Rollins Road, Columbia, Missouri 65211, USA
5 Department of Anatomy, University of Wisconsin-Madison, 1300 University Avenue Madison, Wisconsin 53706, USA
6 Department of Neurology, University of Wisconsin-Madison, 600 North Highland Avenue, Madison, Wisconsin 53792, USA
Spinal muscular atrophy is one of the most common inherited forms of neurological disease leading to infant mortality. Patients have selective loss of lower motor neurons resulting in muscle weakness, paralysis and often death. Although patient fibroblasts have been used extensively to study spinal muscular atrophy, motor neurons have a unique anatomy and physiology which may underlie their vulnerability to the disease process. Here we report the generation of induced pluripotent stem cells from skin fibroblast samples taken from a child with spinal muscular atrophy. These cells expanded robustly in culture, maintained the disease genotype and generated motor neurons that showed selective deficits compared to those derived from the child's unaffected mother. This is the first study to show that human induced pluripotent stem cells can be used to model the specific pathology seen in a genetically inherited disease. As such, it represents a promising resource to study disease mechanisms, screen new drug compounds and develop new therapies.
