Review: Stem cells and gene therapy.

Faris Q Alenzi, Mahmoud Lotfy, Waleed G Tamimi, Richard K H Wyse
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引用次数: 10

Abstract

Both stem cell and gene therapy research are currently the focus of intense research in institutions and companies around the world. Both approaches hold great promise by offering radical new and successful ways of treating debilitating and incurable diseases effectively. Gene therapy is an approach to treat, cure, or ultimately prevent disease by changing the pattern of gene expression. It is mostly experimental, but a number of clinical human trials have already been conducted. Gene therapy can be targeted to somatic or germ cells; the most common vectors are viruses. Scientists manipulate the viral genome and thus introduce therapeutic genes to the target organ. Viruses, in this context, can cause adverse events such as toxicity, immune and inflammatory responses, as well as gene control and targeting issues. Alternative modalities being considered are complexes of DNA with lipids and proteins. Stem cells are primitive cells that have the capacity to self renew as well as to differentiate into 1 or more mature cell types. Pluripotent embryonic stem cells derived from the inner cell mass can develop into more than 200 different cells and differentiate into cells of the 3 germ cell layers. Because of their capacity of unlimited expansion and pluripotency, they are useful in regenerative medicine. Tissue or adult stem cells produce cells specific to the tissue in which they are found. They are relatively unspecialized and predetermined to give rise to specific cell types when they differentiate. The current review provides a summary of our current knowledge of stem cells and gene therapy as well as their clinical implications and related therapeutic options.

综述:干细胞与基因治疗。
干细胞和基因治疗研究目前是世界各地机构和公司的研究热点。这两种方法都有很大的希望,提供了彻底的、新的、成功的方法来有效地治疗衰弱和不治之症。基因治疗是一种通过改变基因表达模式来治疗、治愈或最终预防疾病的方法。它主要是实验性的,但已经进行了一些临床人体试验。基因治疗可以针对体细胞或生殖细胞;最常见的载体是病毒。科学家操纵病毒基因组,从而将治疗基因引入目标器官。在这种情况下,病毒可引起诸如毒性、免疫和炎症反应以及基因控制和靶向问题等不良事件。正在考虑的替代方式是DNA与脂质和蛋白质的复合物。干细胞是一种原始细胞,具有自我更新和分化成一种或多种成熟细胞类型的能力。来源于内层细胞群的多能胚胎干细胞可发育成200多种不同的细胞,并分化为3个生殖细胞层的细胞。由于它们具有无限扩展和多能性,因此在再生医学中非常有用。组织干细胞或成体干细胞产生的细胞对它们所在的组织具有特异性。它们相对来说是非特化的,当它们分化时,它们会产生特定的细胞类型。目前的综述提供了我们目前的干细胞和基因治疗的知识,以及他们的临床意义和相关的治疗方案的总结。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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