Myogenic Cell Transplantation in Genetic and Acquired Diseases of Skeletal Muscle.
Myogenic Cell Transplantation in Genetic and Acquired Diseases of Skeletal Muscle.
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DOI：
10.3389/fgene.2021.702547
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发表时间：
2021
影响因子：
3.7
通讯作者：
Study Group
中科院分区：
文献类型：
--
作者：
Boyer O;Butler-Browne G;Chinoy H;Cossu G;Galli F;Lilleker JB;Magli A;Mouly V;Perlingeiro RCR;Previtali SC;Sampaolesi M;Smeets H;Schoewel-Wolf V;Spuler S;Torrente Y;Van Tienen F;Study Group
本文就肌源性细胞移植治疗先天性和获得性骨骼肌疾病作一综述。关于这个主题已经有一些很好的评论，但它们大多集中在特定的疾病，肌营养不良，特别是杜氏肌营养不良症。最近也有关于细胞移植治疗炎症性肌病、容积性肌肉丧失(VML)(通常使用生物材料)、肌肉量减少和括约肌失禁(主要是尿液，但也包括粪便)的综述。我们认为，在这个阶段，比较在所有这些不同疾病中采用的相同策略将是有用的，以便概述细胞来源、临床前模型、给药途径和结果衡量标准的异同。这反过来可能有助于了解到，到目前为止，哪些共同或特定于疾病的问题限制了细胞移植在这一领域的临床成功，特别是与其他领域相比，如上皮细胞移植。我们还希望，这可能对外地人士在一次审查中获得全面看法有所帮助。对于任何细胞移植程序，在自体和异体细胞之间的选择取决于许多标准，例如细胞的可用性、体外扩增达到所需数量的可能性、对许多但不一定是所有肌肉营养不良患者进行基因纠正的必要性、以及免疫反应，主要是异种的，即使是HLA匹配的细胞，以及少量的治疗性基因产物，可能是患者的抗原。最后，已经进入其他疾病临床实验的诱导多能干细胞衍生品，未来可能会提供一批免疫特权细胞库，供所有患者使用，并在对肌肉营养不良和其他肌肉疾病进行基因纠正后使用。
This article will review myogenic cell transplantation for congenital and acquired diseases of skeletal muscle. There are already a number of excellent reviews on this topic, but they are mostly focused on a specific disease, muscular dystrophies and in particular Duchenne Muscular Dystrophy. There are also recent reviews on cell transplantation for inflammatory myopathies, volumetric muscle loss (VML) (this usually with biomaterials), sarcopenia and sphincter incontinence, mainly urinary but also fecal. We believe it would be useful at this stage, to compare the same strategy as adopted in all these different diseases, in order to outline similarities and differences in cell source, pre-clinical models, administration route, and outcome measures. This in turn may help to understand which common or disease-specific problems have so far limited clinical success of cell transplantation in this area, especially when compared to other fields, such as epithelial cell transplantation. We also hope that this may be useful to people outside the field to get a comprehensive view in a single review. As for any cell transplantation procedure, the choice between autologous and heterologous cells is dictated by a number of criteria, such as cell availability, possibility of in vitro expansion to reach the number required, need for genetic correction for many but not necessarily all muscular dystrophies, and immune reaction, mainly to a heterologous, even if HLA-matched cells and, to a minor extent, to the therapeutic gene product, a possible antigen for the patient. Finally, induced pluripotent stem cell derivatives, that have entered clinical experimentation for other diseases, may in the future offer a bank of immune-privileged cells, available for all patients and after a genetic correction for muscular dystrophies and other myopathies.
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影响因子：
5.5
作者：
Alonso-Martin S;Rochat A;Mademtzoglou D;Morais J;de Reyniès A;Auradé F;Chang TH;Zammit PS;Relaix F
通讯作者：
Relaix F
影响因子：
7.3
作者：
Alston, Charlotte L.;Rocha, Mariana C.;Lax, Nichola Z.;Turnbull, Doug M.;Taylor, Robert W.
通讯作者：
Taylor, Robert W.
DOI：
10.1007/s004290050237
发表时间：
1999-05-01
期刊：
ANATOMY AND EMBRYOLOGY
影响因子：
--
作者：
Bittner, RE;Schöfer, C;Wachtler, F
通讯作者：
Wachtler, F
影响因子：
14.5
作者：
CARPENTER, S;KARPATI, G
通讯作者：
KARPATI, G
影响因子：
23.9
作者：
Benchaouir, Rachid;Meregalli, Mirella;Torrente, Yvan
通讯作者：
Torrente, Yvan