Résumé :
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The lack of existing models of human pathologic tissues has rendered many important questions in disease pathogenesis inaccessible until now. Disease-specific human pluripotent stem cells, from embryonic origin or more recently derived from reprogramming somatic cells, offer the unique opportunity to have access to a large spectrum of disease-specific cell models. Due to their ability of self renewal and differentiation into various tissues affected in each pathological condition, the development of these human disease-specific pluripotent stem cells should provide new insights in pathological mechanisms implicated in human diseases and subsequently permit the development of new therapeutic strategies. Validating this original concept, we recently demonstrated that PGD-derived hES cells and derivatives which, express the causal mutation implicated in the Myotonic Dystrophy type 1 (DM1), provide new insights into the neuropathological mechanisms underlying this monogenic disease and can be used to identify new therapeutic strategies.
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