A step closer toward therapies for p63-related disorders

Figures & data

Figure 1. Description of the two cellular models used to demonstrate the effect of the small compound APR246/PRIMA-1^MET on epithelial commitment and differentiation. (1) p63 is required for proper epidermal and corneal commitment of ectodermal progenitors during mammalian development¹⁹ and epidermal stem cells/progenitors self-renewal and differentiation.²⁰(2) Dermal fibroblasts (or skin keratinocytes) can be reprogrammed into embryonic-like cells called induced pluripotent stem cells (iPSC) in vitro.²¹ EEC-iPSC derived from reprogramming of fibroblasts are unable to differentiate into corneal epithelial cells properly and treatment with APR246/PRIMA-1^MET improved corneal commitment. (3) Keratinocytes isolated from EEC patients defects in epidermal differentiation and stratification, which are partially restored with APR246/PRIMA-1^MET treatment.

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