基于多能干细胞的肌肉疾病治疗方法。
Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies.
机构信息
Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA; Stem Cell Institute, University of Minnesota, Minneapolis, MN, USA.
出版信息
Trends Mol Med. 2019 Sep;25(9):803-816. doi: 10.1016/j.molmed.2019.07.004.
Abstract
Pluripotent stem cells (PSCs) represent an attractive cell source for treating muscular dystrophies (MDs) since they easily allow for the generation of large numbers of highly regenerative myogenic progenitors. Using reprogramming technology, patient-specific PSCs have been derived for several types of MDs, and genome editing has allowed correction of mutations, opening the opportunity for their therapeutic application in an autologous transplantation setting. However, there has been limited progress on preclinical studies that validate the therapeutic potential of these gene corrected PSC-derived myogenic progenitors. In this review, we highlight the major research advances, challenges, and future prospects towards the development of PSC-based therapeutics for MDs.
摘要
多能干细胞（PSCs）是治疗肌肉萎缩症（MDs）的有吸引力的细胞来源，因为它们可以轻松地产生大量高度再生的成肌祖细胞。利用重编程技术，已经从几种类型的 MD 患者中获得了患者特异性 PSCs，并且基因组编辑允许纠正突变，为它们在自体移植环境中的治疗应用开辟了机会。然而，在验证这些经过基因修正的 PSC 衍生的成肌祖细胞的治疗潜力的临床前研究方面进展有限。在这篇综述中，我们强调了在开发基于 PSC 的 MD 治疗方法方面的主要研究进展、挑战和未来前景。
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