Sounds like a catchy song title…………..but it’s actually an interesting proof-of-principle study published this week in the Orphanet Journal of Rare Diseases, by Edward Hsiao and his colleagues at UCSF.
“Induced pluripotent stem cells from patients with human fibrodysplasia ossificans progressiva show increased mineralization and cartilage formation.”
Open Access article available here.
The authors have generated human induced pluripotent stem cells (iPS cells) from both normal and Fibrodysplasia Ossificans Progressiva (FOP) patient fibroblasts. These iPS cells were then induced to a chondrocyte-like phenotype for disease mechanism studies.
The authors conclude that this study provides a proof-of-concept for using human iPS cell models to understand human skeletal disorders.
Moreover, they conclude that:-
“Human iPS cells will be a valuable tool that reflects the diversity of patients we see in the clinic since patients, and thus, their iPS cells show clinical variability, genetic background effects, and epigenetic influences……….combined with translational genetic studies and correlations with murine models, provides exciting venues for new strategies to understand the stages of normal and pathologic human skeletal development.”
One important task of EU_FP7 SYBIL is to exploit new biological resources and technologies to generate iPS cell lines from rare disease patients and differentiate them into skeletal-relevant cell lines.
More specifically, in Work Package 2 human iPS cells will be generated from patient cells such as dermal fibroblasts, blood cells or urine derived cells. SYBIL partners University of Manchester, Institute of Genetic and Biomedical Research and Evercyte will be taking a leading role in this exciting and cutting edge translational research.