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首页> 外文期刊>Expert opinion on biological therapy >Non-viral episomal modification of cells using S/MAR elements.
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Non-viral episomal modification of cells using S/MAR elements.

机译：使用S / MAR元件对细胞进行非病毒游离修饰。

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INTRODUCTION: The early potential of gene therapy is slowly becoming realized following the recent treatment of patients with severe combined immunodeficiency and ocular diseases. However at present the field of gene therapy is tempered by the toxicity issues, mainly that of the integrated retroviral vector used in most trials which led to oncogenesis in several of the treated patients. The development of safer, alternative vectors is therefore vital for further progress in this field, in particular vectors which remain episomal and are therefore less genotoxic. One such unique class of vectors are those based on scaffold matrix attachment regions (S/MARs) elements, which are maintained extra-chromosomally and replicate in vitro and in vivo. AREAS COVERED: The overview here describes the most relevant studies utilizing the S/MAR element to episomally modify mammalian cells and tissues with a particular focus on liver tissue, as well as the brain, the muscle, the eye, cancer cells, embryonic cells and neonatal mice. For this purpose, recently published data in these areas (mainly articles published between 2000 and 2010) are reviewed. EXPERT OPINION: The utilisation of vectors harbouring an S/MAR element is an efficient, safe and cost-effective way to episomally modify mammalian cells.
机译：简介：在对患有严重免疫缺陷和眼部疾病的患者进行近期治疗之后，基因治疗的早期潜力正逐渐被意识到。然而，目前基因治疗领域受到毒性问题的影响，主要是大多数试验中使用的整合逆转录病毒载体的毒性问题，该逆转录病毒载体导致了一些治疗患者的肿瘤发生。因此，开发更安全，替代性的载体对于该领域的进一步发展至关重要，尤其是保持游离状态且因此遗传毒性较小的载体。一种此类独特的载体是基于支架基质附着区（S / MARs）元件的载体，它们在染色体外被维持并在体外和体内复制。覆盖的区域：这里的概述描述了利用S / MAR元素对哺乳动物细胞和组织进行游离修饰的最相关研究，特别关注肝脏组织以及大脑，肌肉，眼睛，癌细胞，胚胎细胞和新生小鼠。为此，我们回顾了这些领域最近发表的数据（主要是2000年至2010年之间发表的文章）。专家意见：利用带有S / MAR元件的载体是游离修饰哺乳动物细胞的一种有效，安全且经济高效的方法。

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