**Research on Skeletal Muscle Diseases Using Pluripotent Stem Cells**

Lorena de Oñate, Elena Garreta, Carolina Tarantino, Elena Martínez, Encarnación Capilla, Isabel Navarro, Joaquín Gutiérrez, Josep Samitier, Josep Maria Campistol, Pura Muñoz-Cánovas and Nuria Montserrat

Additional information is available at the end of the chapter

http://dx.doi.org/10.5772/60902

#### **Abstract**

The generation of induced pluripotent stem cells (iPSCs), especially the generation of patient-derived pluripotent stem cells (PSCs) suitable for disease modelling *in vitro*, opens the door for the potential translation of stem-cell related studies into the clinic. Successful replacement, or augmentation, of the function of damaged cells by patientderived differentiated stem cells would provide a novel cell-based therapy for skeletal muscle-related diseases. Since iPSCs resemble human embryonic stem cells (hESCs)in their ability to generate cells of the three germ layers, patient-specific iPSCs offer definitive solutions for the ethical and histo-incompatibility issues related to hESCs. IndeedhumaniPSC(hiPSC)-basedautologous transplantationisheraldedas thefuture ofregenerative medicine.Interestingly, during the last years intense research has been published on disease-specific hiPSCs derivation and differentiation into relevant tissues/organsprovidingauniquescenarioformodellingdiseaseprogression,toscreen patient-specific drugs and enabling immunosupression-free cell replacement therapies. Here, we revise the most relevant findings in skeletal muscle differentia‐ tion using mouse and human PSCs. Finally and in an effort to bring iPSC technology to the daily routine of the laboratory, we provide two different protocols for the generation of patient-derived iPSCs.

**Keywords:** Pluripotent stem cells, Myogenic differentiation, Disease modelling, Pa‐ tient-specific induced pluripotent stem cells, Muscular dystrophy

© 2015 The Author(s). Licensee InTech. This chapter is distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
