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说明：原文来自http://www.medicalnewstoday.com/articles/221226.php; `% D/ z7 {( d7 T m/ F/ v0 ~9 D
由干细胞之家新闻小组成员laputave翻译（转帖请注明）
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& j% @" f) O0 D; g) Z V翻译正文：
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& g3 F# w- c' I2 r[标题]经重编程与基因修复后的患者自身细胞有望应用于治疗6 @1 s2 _/ T7 x+ }9 T# ~/ ~ e
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通过确定修复细胞遗传缺陷的过程并不会在实质上增加致癌基因突变的可能性的研究，来自Morgridge研究院，威斯康星大学，加利福尼亚大学和Wicell研究院的科学家们将基因治疗像临床应用又推进了一步。
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6 K$ d2 {0 z( M3 K他们的工作成果预订发表在4月4日的美国国家科学院院刊在线版周刊上，并获得了美国抗盲基金会下设的Wynn-Gund转化成果奖。该研究表明，已经转变为修复基因型的人类IPSC可以培养出无原初疾病的后代细胞。然而，虽然基因修复本身并没有增加细胞遗传的不稳定性及可观测突变量，但这项研究同样也补充了一些关于IPSC本身就携带有大量遗传突变的近期发现。
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# \+ j. _; K7 I! [1 ESara Howden说：“这项研究表明基因修复可以应用到临床治疗中去，并且首次证实了通过同源重组修复患者细胞的缺陷基因的方法是可行的。” Sara Howden是这项工作的第一作者，她在Morgridge研究院James Thomson的实验室做博士后研究助理。
" v& T. c% i/ u4 l与人类ESC一样，IPSC可以分化形成人体220种成熟细胞类型中的任何一种。IPSC是皮肤或其他高度分化的细胞通过与基因或蛋白复合物相接触发生重编程，从而恢复其多能性的一类细胞。
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尽管IPSC来源于患者自身的细胞，但与人类ESC相比，IPSC有许多临床优势，IPSC也避免了免疫排斥反应。尽管如此，科学家们仍然致力于IPSC与人类ESC之间细微差异的研究，包括IPSC更高的基因突变率和其仍然保存有原来所属谱系的“记忆”的证据。! s, c( F u( E
在许多遗传性和获得性疾病如亨廷顿舞蹈症，退行性视网膜病变和糖尿病的治疗方面，有望应用IPSC进行基因治疗。该项研究中有一名患者患有一种被称作回旋性视网膜萎缩的退行性视网膜疾病，这种疾病的症状是渐进性的视敏度降低和夜间视力减退，直至最终全部丧失视力。虽然遗传性视网膜病变和糖尿病等疾病为以IPSC为基础的移植疗法提供治疗靶点，但研究者们也注意到了IPSC常出现遗传突变及其潜在的致癌性。
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4 y& ^" F+ T4 V! O1 Y; G' _注明：个人翻译，理解不准确的语句还望大家批评指正。
" Y3 f0 @! p" ^0 p( W) W+ V[个人首次翻译，不妥之处还请各位大师不吝赐教，晚生谢谢各位 ]: [2 y# J3 d0 z3 Q4 P6 B
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Patient's Own Cells May Hold Therapeutic Promise After Reprogramming, Gene Correction* a0 F9 H2 B& `5 z" C! m
Main Category: Stem Cell Research
9 @2 W% C/ x5 O8 p* w0 ~2 F- MArticle Date: 05 Apr 2011 - 0:00 PDT
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) `# y$ K* t, J6 f7 L; ]Scientists from the Morgridge Institute for Research, the University of Wisconsin-Madison, the University of California and the WiCell Research Institute moved gene therapy one step closer to clinical reality by determining that the process of correcting a genetic defect does not substantially increase the number of potentially cancer-causing mutations in induced pluripotent stem cells.
8 K* \7 Z" D8 U. T2 X/ w$ mTheir work, scheduled for publication the week of April 4 in the online edition of the journal Proceedings of the National Academy of Sciences and funded by a Wynn-Gund Translational Award from the Foundation Fighting Blindness, suggests that human induced pluripotent stem cells altered to correct a genetic defect may be cultured into subsequent generations of cells that remain free of the initial disease. However, although the gene correction itself does not increase the instability or the number of observed mutations in the cells, the study reinforced other recent findings that induced pluripotent stem cells themselves carry a significant number of genetic mutations.
3 T: f2 z$ P% N: g, \"This study showed that the process of gene correction is compatible with therapeutic use," says Sara Howden, primary author of the study, who serves as a postdoctoral research associate in James Thomson's lab at the Morgridge Institute for Research. "It also was the first to demonstrate that correction of a defective gene in patient-derived cells via homologous recombination is possible."
! u! o6 r) E6 z7 FLike human embryonic stem cells, induced pluripotent stem cells can become any of the 220 mature cell types in the human body. Induced pluripotent stem cells are created when skin or other mature cells are reprogrammed to a pluripotent state through exposure to select combinations of genes or proteins.. M! S4 W" g1 b" [2 A; u; U
Since they can be derived from a patient's own cells, induced pluripotent stem cells may offer some clinical advantages over human embryonic stem cells by avoiding problems with rejection. However, scientists are still working to understand subtle differences between human embryonic and induced pluripotent stem cells, including a higher rate of genetic mutations among the induced pluripotent cells and evidence that the cells may retain some "memory" of their previous lineage.
6 E( @& [9 v2 l/ Y$ m" xGene therapy using induced pluripotent stem cells holds promise for treating many inherited and acquired diseases such as Huntington's disease, degenerative retinal disease or diabetes. The patient in this study suffers from a degenerative eye disease known as gyrate atrophy, which is characterized by progressive loss of visual acuity and night vision leading to eventual blindness. While diseases such as genetic retinal disorders and diabetes offer attractive targets for induced pluripotent stem cell-based transplant therapies, concerns have been raised over the commonly occurring mutations in the cells and their potential to become cancerous.
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发表于 2011-4-7 13:45
发表于 2011-4-7 15:05
不错！！
