Research on Skeletal Muscle Diseases Using Pluripotent Stem Cells

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 augmenta...

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Detalles Bibliográficos
Autores: Oñate, Lorena de, Garreta, Elena, Tarantino, Carolina, Martínez Fraiz, Elena, Capilla Campos, Encarnación, Navarro Álvarez, Isabel, Gutiérrez Fruitós, Joaquín, Samitier i Martí, Josep, Campistol Plana, Josep M., Muñoz Cánoves, Pura, 1962-, Montserrat Pulido, Núria
Tipo de recurso: capítulo de libro
Estado:Versión publicada
Fecha de publicación:2015
País:España
Institución:Universidad de Barcelona
Repositorio:Dipòsit Digital de la UB
OAI Identifier:oai:diposit.ub.edu:2445/175835
Acceso en línea:https://hdl.handle.net/2445/175835
Access Level:acceso abierto
Palabra clave:Cèl·lules mare
Distròfia muscular
Stem cells
Muscular dystrophy
Descripción
Sumario: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. Indeed human iPSC (hiPSC)-based autologous transplantation is heralded as the future of regenerative medicine. Interestingly, during the last years intense research has been published on disease-specific hiPSCs derivation and differentiation into relevant tissues/organs providing a unique scenario for modelling disease progression, to screen patient-specific drugs and enabling immunosupression-free cell replacement therapies. Here, we revise the most relevant findings in skeletal muscle differentiation 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.