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Research on Skeletal Muscle Diseases Using Pluripotent Stem Cells

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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.

# metadata.dc.source[].value
Llibres / Capítols de llibre (Biologia Cel·lular, Fisiologia i Immunologia)

# metadata.dc.title[].value
Research on Skeletal Muscle Diseases Using Pluripotent Stem Cells