Fate Therapeutics Inc. San Diego, CA, USA.
Front Cell Dev Biol. 2015 May 13;3:29. doi: 10.3389/fcell.2015.00029. eCollection 2015.
Human pluripotent stem cells (hPSCs) possess unlimited proliferative potential while maintaining the ability to differentiate into any cell type including skeletal muscle cells (SMCs). hPSCs are amenable to genetic editing and can be derived from patient somatic cells, and thus represent a promising option for cell therapies for the treatment of degenerative diseases such as muscular dystrophies. There are unresolved challenges however associated with the derivation and scale-up of hPSCs and generation of differentiated cells in large quantity and high purity. Reported myogenic differentiation protocols are long, require cell sorting and/or rely on ectopic expression of myogenic master regulators. More recent advances have been made with the application of small molecules to enhance the myogenic differentiation efficiency and the identification of more selective markers for the enrichment of myogenic progenitors with enhanced regenerative potential. Here we review the field of myogenic differentiation and highlight areas requiring further research.
人类多能干细胞（hPSCs）具有无限增殖的潜力，同时保持分化为包括骨骼肌细胞（SMCs）在内的任何细胞类型的能力。hPSCs 易于进行基因编辑，并且可以从患者的体细胞中获得，因此代表了治疗退行性疾病（如肌肉营养不良症）的细胞治疗的有前途的选择。然而，与 hPSCs 的衍生和规模化以及大量和高纯度分化细胞的产生相关的仍存在未解决的挑战。报道的成肌分化方案时间长，需要细胞分选和/或依赖于肌形成主调控因子的异位表达。随着小分子的应用，成肌分化效率得到了提高，并且鉴定出了更具选择性的标记物，用于富集具有增强再生潜能的成肌祖细胞。在这里，我们综述了成肌分化领域，并强调了需要进一步研究的领域。
