摘要
人类多能干细胞代表着基于细胞疗法和再生医学的唯一来源。这些细胞的内在特性,如易于获取和无限增殖能力,使其能克服传统成体干细胞的一些局限性。此外,源于患者特异性诱导多能干(iPS)细胞与当前基因修饰方法相结合,使其用于某些遗传性疾病的自体细胞治疗。迄今为止,由于缺乏对患者有效的治疗方案,并鉴于最近临床前研究中所产生的乐观结果,肌肉萎缩症被认为是一个很适合诊治疾病。然而,存在的一些障碍仍有待克服,有些方法也需进一步优化,如可能的遗传不稳定性和通过载体-转基因方法使其高效分化成肌肉细胞等。此外,移植和临床前肌肉再生功能模型的作用,需要在临床转化前进行仔细评估。本文就最近开发的多能干细胞诱导产生肌祖细胞的先进方法,以及基因疗法和基因剪接法(ZFNs, TALENs 和 CRISPR/Cas9)等进行了概述,此外,也讨论了需要解决的主要问题,为了能成功进行临床转化,在骨骼肌疾病自体移植试验中从遗传学角度来纠正患者特异性多能干细胞。
关键词: 细胞治疗,核酸酶设计师 ,胚胎干细胞,基因治疗,诱导多能干细胞(iPS),肌肉干细胞,肌肉萎缩症,再生医学。
Current Gene Therapy
Title:Pluripotent Stem Cells for Gene Therapy of Degenerative Muscle Diseases
Volume: 15 Issue: 4
Author(s): Mariana Loperfido, Heather B. Steele-Stallard, Francesco Saverio Tedesco and Thierry VandenDriessche
Affiliation:
关键词: 细胞治疗,核酸酶设计师 ,胚胎干细胞,基因治疗,诱导多能干细胞(iPS),肌肉干细胞,肌肉萎缩症,再生医学。
摘要: Human pluripotent stem cells represent a unique source for cell-based therapies and regenerative medicine. The intrinsic features of these cells such as their easy accessibility and their capacity to be expanded indefinitely overcome some limitations of conventional adult stem cells. Furthermore, the possibility to derive patient-specific induced pluripotent stem (iPS) cells in combination with the current development of gene modification methods could be used for autologous cell therapies of some genetic diseases. In particular, muscular dystrophies are considered to be a good candidate due to the lack of efficacious therapeutic treatments for patients to date, and in view of the encouraging results arising from recent preclinical studies. Some hurdles, including possible genetic instability and their efficient differentiation into muscle progenitors through vector/transgene-free methods have still to be overcome or need further optimization. Additionally, engraftment and functional contribution to muscle regeneration in pre-clinical models need to be carefully assessed before clinical translation. This review offers a summary of the advanced methods recently developed to derive muscle progenitors from pluripotent stem cells, as well as gene therapy by gene addition and gene editing methods using ZFNs, TALENs or CRISPR/Cas9. We have also discussed the main issues that need to be addressed for successful clinical translation of genetically corrected patient-specific pluripotent stem cells in autologous transplantation trials for skeletal muscle disorders.
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Mariana Loperfido, Heather B. Steele-Stallard, Francesco Saverio Tedesco and Thierry VandenDriessche , Pluripotent Stem Cells for Gene Therapy of Degenerative Muscle Diseases, Current Gene Therapy 2015; 15 (4) . https://dx.doi.org/10.2174/1566523215666150630121207
DOI https://dx.doi.org/10.2174/1566523215666150630121207 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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