Department of Medicine and Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA.
Transl Res. 2013 Apr;161(4):284-92. doi: 10.1016/j.trsl.2013.01.001. Epub 2013 Jan 23.
Human pluripotent stem cells represent an accessible cell source for novel cell-based clinical research and therapies. With the realization of induced pluripotent stem cells (iPSCs), it is possible to produce almost any desired cell type from any patient's cells. Current developments in gene modification methods have opened the possibility for creating genetically corrected human iPSCs for certain genetic diseases that could be used later in autologous transplantation. Promising preclinical studies have demonstrated correction of disease-causing mutations in a number of hematological, neuronal, and muscular disorders. This review aims to summarize these recent advances with a focus on iPSC generation techniques, as well as gene modification methods. We will then further discuss some of the main obstacles remaining to be overcome before successful application of human pluripotent stem cell-based therapy arrives in the clinic and what the future of stem cell research may look like.
人类多能干细胞是一种易于获取的细胞来源，可用于新型基于细胞的临床研究和治疗。随着诱导多能干细胞（iPSC）的实现，几乎可以从任何患者的细胞中产生任何所需的细胞类型。目前基因修饰方法的发展为某些遗传性疾病创造了基因校正的人类 iPSC 的可能性，这些细胞可随后用于自体移植。有前途的临床前研究已经证明了在许多血液、神经和肌肉疾病中纠正致病突变的可能性。本综述旨在总结这些最新进展，重点介绍 iPSC 生成技术以及基因修饰方法。然后，我们将进一步讨论在基于人类多能干细胞的治疗成功应用于临床之前仍然需要克服的一些主要障碍，以及干细胞研究的未来可能会是什么样子。
