Skuk Daniel, Tremblay Jacques P
Unité de recherche en Génétique humaine, Centre de Recherche du Centre Hospitalier de l'Université Laval, Québec, Canada.
Curr Opin Rheumatol. 2003 Nov;15(6):723-9. doi: 10.1097/00002281-200311000-00007.
Cell therapies for inherited myopathies are based on the implantation of normal or genetically corrected myogenic cells into the body. This review summarizes the recent progress in this field, systematized according to the factors important for success.
In the choice of donor cells, myoblasts derived from satellite cells remain the best choice. Some studies on the population of muscle-derived stem cells in mice suggested that these cells may have some advantages over myoblasts; however, no results supporting this advantage have been presented in a primate model. Recent studies on bone marrow transplantation as a systemic source of myogenic precursors for the treatment of myopathies were disappointing. Concerning donor cell delivery, intramuscular myoblast injection remains the only way that can significantly introduce exogenous myogenic cells into the muscles. A recent study in primates showed some parameters of myoblast injection that could be useful in the human. Progress was made in mice to understand the factors that could favor the migration of the donor myoblasts in the host muscles. Concerning donor cell survival, analysis of immune cell infiltration dynamics allowed a better understanding of the factors implicated in early donor cell death. Progress was made on the control of acute rejection for myoblast transplantation in primates. So far, few mouse experiments have advanced the field of tolerance induction toward myogenic cells.
Myoblast transplantation (intramuscular injection of satellite cell-derived myoblasts) currently remains the only cell-based therapy that has produced promising results in the context of a preclinical model such as the nonhuman primate.
遗传性肌病的细胞疗法是基于将正常或基因校正的成肌细胞植入体内。本综述总结了该领域的最新进展，并根据对成功至关重要的因素进行了系统整理。
在供体细胞的选择上，源自卫星细胞的成肌细胞仍是最佳选择。一些关于小鼠肌肉衍生干细胞群体的研究表明，这些细胞可能比成肌细胞具有某些优势；然而，在灵长类动物模型中尚未有支持这一优势的结果。最近关于骨髓移植作为治疗肌病的成肌前体系统来源的研究令人失望。关于供体细胞的递送，肌肉内注射成肌细胞仍然是能够将外源性成肌细胞显著引入肌肉的唯一方法。最近一项针对灵长类动物的研究显示了一些成肌细胞注射参数，这些参数可能对人类有用。在小鼠研究中取得了进展，以了解有利于供体成肌细胞在宿主肌肉中迁移的因素。关于供体细胞的存活，对免疫细胞浸润动态的分析有助于更好地理解与早期供体细胞死亡相关的因素。在灵长类动物中，成肌细胞移植的急性排斥控制方面取得了进展。到目前为止，很少有小鼠实验推动了对成肌细胞耐受性诱导领域的研究。
成肌细胞移植（肌肉内注射卫星细胞衍生的成肌细胞）目前仍然是唯一一种在非人类灵长类动物等临床前模型中产生了有前景结果的基于细胞的疗法。
