CHUQ Research Center - CHUL, Neurosciences Division - Human Genetics, 2705 Boulevard Laurier, Quebec, Quebec G1V 4G2, Canada.
Expert Opin Biol Ther. 2011 Mar;11(3):359-74. doi: 10.1517/14712598.2011.548800. Epub 2011 Jan 5.
Myopathies produce deficits in skeletal muscle function and, in some cases, literally progressive loss of skeletal muscles. The transplantation of cells able to differentiate into myofibers is an experimental strategy for the potential treatment of some of these diseases.
Among the two routes used to deliver cells to skeletal muscles, that is intramuscular and intravascular, this paper focuses on the intramuscular route due to our expertise and because it is the most used in animal experiments and the only tested so far in humans. Given the absence of recent reviews about clinical observations and the profusion based on mouse results, this review prioritizes observations made in humans and non-human primates. The review provides a vision of cell transplantation in myology centered on what can be learned from clinical trials and from preclinical studies in non-human primates and leading mouse studies.
Experiments on myogenic cell transplantation in mice are essential to quickly identify potential treatments, but studies showing the possibility to scale up the methods in large mammals are indispensable to determine their applicability in humans and to design clinical protocols.
肌病会导致骨骼肌功能缺陷，在某些情况下，甚至会导致骨骼肌逐渐丧失。将能够分化为肌纤维的细胞移植是治疗某些此类疾病的一种实验策略。
在将细胞递送到骨骼肌的两种途径中，即肌内途径和血管内途径，本文侧重于肌内途径，这是因为我们在该领域具有专业知识，并且该途径在动物实验中应用最广泛，迄今为止仅在人体中进行了测试。鉴于最近没有关于临床观察的综述，并且基于小鼠结果的综述泛滥，因此本综述优先考虑了在人体和非人类灵长类动物中观察到的结果。该综述提供了一个以从临床试验和非人类灵长类动物的临床前研究中获得的知识为中心的肌细胞移植的观点，这些研究引领了小鼠研究。
在小鼠中进行肌源性细胞移植的实验对于快速确定潜在的治疗方法至关重要，但是，显示出将方法在大型哺乳动物中放大的可能性的研究对于确定其在人体中的适用性以及设计临床方案是不可或缺的。
