（圖片來源：Kit Rodolfa和哈佛大學(xué)的John Dimos）
據(jù)7月31日的《科學(xué)》（Science）雜志報(bào)道說,，研究人員朝著應(yīng)用誘導(dǎo)多能干細(xì)胞（或稱“iPS”細(xì)胞）來治療疾病的目標(biāo)又邁出了重要的一步,。研究人員從來自2位罹患肌萎縮性脊髓側(cè)索硬化癥（ALS）這種神經(jīng)退行性病變的老年病人的皮膚采樣中制造出了iPS。而且，他們用這些iPS 細(xì)胞發(fā)展出了看來像是健康的運(yùn)動(dòng)神經(jīng)元細(xì)胞,。較早的研究顯示,，iPS 細(xì)胞就像是多能胚胎干細(xì)胞那樣可以演變?yōu)槎喾N類型的細(xì)胞，而這些iPS 可以從健康的捐贈(zèng)者的細(xì)胞中產(chǎn)生,。但是,，這種技術(shù)是否也適用于來自罹患慢性疾病的老年人則仍然是一個(gè)懸而未決的問題。
John Dimos 及其同事開始對(duì)來自兩位罹患遺傳形式的ALS 病患的皮膚細(xì)胞進(jìn)行實(shí)驗(yàn),，并通過加入四種基因?qū)⑦@些細(xì)胞轉(zhuǎn)變回iPS 細(xì)胞，而這四種基因是研究人員先前用來將細(xì)胞程序進(jìn)行重新設(shè)定使其演變?yōu)閕PS 狀態(tài)的基因,。接著,，研究人員將來自其中一名病患的iPS 細(xì)胞沉浸在多種信號(hào)分子中，設(shè)法使這些細(xì)胞成為看上去像是運(yùn)動(dòng)神經(jīng)元的細(xì)胞,，而運(yùn)動(dòng)神經(jīng)元是在ALS 中遭損害的細(xì)胞,。這一做法的終極希望是：用像這樣的iPS 來制造在遺傳上相匹配的健康細(xì)胞，并用其取代病變的細(xì)胞,。但是,，在將這種方法安全地用于人身上之前，仍然還有重大的障礙需要克服,。與此同時(shí),，病患特異性的iPS 細(xì)胞將是研究ALS 樣疾病發(fā)生機(jī)理的重要工具。在大多數(shù)的情況下,，ALS是遺傳與環(huán)境因子間復(fù)雜的相互作用的結(jié)果,，這使得在細(xì)胞培養(yǎng)中來研究這種疾病變得非常困難。但是,，來自有遺傳變異（這些變異使得變異基因攜帶者容易罹患該種疾?。┑牟∪说膇PS 細(xì)胞則恰好攜帶著個(gè)體病人中與該疾病有關(guān)的“眾多”的遺傳信息。（生物谷Bioon.com）
生物谷推薦原始出處：
Science,，DOI: 10.1126/science.1158799,，John T. Dimos，Kevin Eggan
Induced Pluripotent Stem Cells Generated from Patients with ALS Can Be Differentiated into Motor Neurons
John T. Dimos 1, Kit T. Rodolfa 2, Kathy K. Niakan 1, Laurin M. Weisenthal 1, Hiroshi Mitsumoto 3, Wendy Chung 4, Gist F. Croft 5, Genevieve Saphier 1, Rudy Leibel 6, Robin Goland 7, Hynek Wichterle 5, Christopher E. Henderson 5, Kevin Eggan 1*
1 Harvard Stem Cell Institute, Stowers Medical Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA.
2 Harvard Stem Cell Institute, Stowers Medical Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA.
3 Eleanor and Lou Gehrig MDA-ALS Research Center, Neurological Institute, Columbia University Medical Center, New York, NY 10032, USA.; Center for Motor Neuron Biology and Disease, Columbia University Medical Center, New York, NY 10032, USA.
4 Center for Motor Neuron Biology and Disease, Columbia University Medical Center, New York, NY 10032, USA.; Division of Molecular Genetics and Naomi Barrie Diabetes Center, College of Physicians and Surgeons, Columbia University, New York, NY 10032, USA.
5 Center for Motor Neuron Biology and Disease, Columbia University Medical Center, New York, NY 10032, USA.; Departments of Pathology, Neurology and Neuroscience, Columbia University Medical Center, New York, NY 10032, USA.
6 Division of Molecular Genetics and Naomi Barrie Diabetes Center, College of Physicians and Surgeons, Columbia University, New York, NY 10032, USA.
7 Department of Medicine and Naomi Barrie Diabetes Center, Columbia University Medical Center, New York, NY 10032, USA.
* To whom correspondence should be addressed.
Kevin Eggan , E-mail: [email protected]
These authors contributed equally to this work.
The generation of pluripotent stem cells from an individual patient would enable the large-scale production of the cell-types affected by that patient’s disease. These cells could in turn be used for disease modeling, drug discovery, and eventually autologous cell-replacement therapies. Although recent studies have demonstrated the reprogramming of human fibroblasts to a pluripotent state, it remains unclear whether these induced pluripotent stem (iPS) cells can be produced directly from elderly patients with chronic disease. We have generated iPS cells from an 82-year-old woman diagnosed with a familial form of amyotrophic lateral sclerosis (ALS). These patient-specific iPS cells possess properties of embryonic stem cells and were successfully directed to differentiate into motor neurons, the cell type destroyed in ALS.
