美國(guó)威斯康辛大學(xué)教授詹姆斯·湯姆森的科研小組日前成功地在試管內(nèi)利用萬(wàn)能細(xì)胞再現(xiàn)了疾病過程,。該小組用重癥神經(jīng)疾病患者的皮膚細(xì)胞培養(yǎng)出人工多功能干細(xì)胞(iPS細(xì)胞),，將這些iPS細(xì)胞培育為神經(jīng)細(xì)胞后，在試管內(nèi)成功再現(xiàn)了神經(jīng)細(xì)胞因疾病死亡的過程。
這是世界首例使用患者iPS細(xì)胞重現(xiàn)病癥的成功嘗試。科研小組在英國(guó)科學(xué)雜志《自然》上發(fā)表了該成果，預(yù)計(jì)今后可廣泛應(yīng)用于探明病因,、研發(fā)新藥等領(lǐng)域。
科研小組表示“隨著時(shí)間推移成功觀測(cè)到了神經(jīng)細(xì)胞出現(xiàn)的異常變化,。在患者體內(nèi)難以觀測(cè)的病變可以在試管內(nèi)清晰再現(xiàn)了”,。
日本iPS細(xì)胞專家、國(guó)立成育醫(yī)療中心研究所室長(zhǎng)阿久津英憲表示,，該成果對(duì)iPS細(xì)胞在治療疾病,、藥物研發(fā)領(lǐng)域中的現(xiàn)實(shí)應(yīng)用邁進(jìn)了一大步。曾有專家擔(dān)心“重現(xiàn)病癥會(huì)很難”,，而該成果體現(xiàn)了研究的飛速發(fā)展,。用iPS細(xì)胞可無限度繁殖病變細(xì)胞用于研究，此意義實(shí)屬非凡,。（生物谷Bioon.com）
生物谷推薦原始出處：
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.
