A Perspective on the Potential of Human iPS Cell-Based Therapies for Muscular Dystrophies: Advancements So Far and Hurdles to Overcome

Pluripotent stem cells [1,2] are well-known for their unique self-renewal and differentiation capabilities, which make these cells very favorable for cell-based therapeutic applications in degenerative disorders such as muscular dystrophies. Major concerns associated with embryonic stem (ES) cells, such as immunological compatibility and ethical considerations had limited their clinical applications [3]. However, the technology of reprogramming somatic cells into induced pluripotent stem (iPS) cells provides a new breath of hope for a potential therapeutic application in incurable diseases [4–6]. iPS cells have been generated initially from mouse and later from human somatic cells by introduction of the four reprogramming transcription factors using retroviral vectors. More recently, this has been accomplished by using non-integrating vectors [7,8], which is much safer as the associated risk of insertional mutagenesis is significantly reduced. Consequently, by proper differentiation of iPS cells into tissue specific progenitors, one can foresee the therapeutic application of iPS cells in a relatively near future, upon extensive testing for the safety of these cell preparations is achieved.