Alessia Secone Seconetti

TL;DR: It is shown that autologous transgenic keratinocyte cultures regenerated an entire, fully functional epidermis on a seven-year-old child suffering from a devastating, life-threatening form of JEB, providing a blueprint that can be applied to other stem cell-mediated combined ex vivo cell and gene therapies.

TL;DR: It is shown that dysregulation of the YAP and TAZ pathway underpins epidermal stem cell depletion in JEB, and this work has important clinical implication for ex vivo gene therapy of JEB.

TL;DR: This review will specifically address the few combined cell and gene therapy strategies that made it to the clinics, and what can be envisioned for the future is a patient-oriented strategy, built on the specific features of the individual in need.

TL;DR: In this paper, single-cell transcriptome analysis of primary human epidermal cultures identifies categories of genes clearly distinguishing the different keratinocyte clonal types, which are hierarchically organized along a continuous, mainly linear trajectory showing that stem cells sequentially generate progenitors producing terminally differentiated cells.