Global gene expression in patient keratinocytes and patient-specific iPS-derived keratinocytes

ABSTRACT: Patients with recessive dystrophic epidermolysis bullosa (RDEB) lack functional Type VII collagen and suffer severe blistering and chronic wounds that ultimately lead to infection and development of lethal squamous cell carcinoma (SCC). The discovery of induced pluripotent stem cells (iPSCs) and the ability to edit the genome bring the possibility to provide definitive genetic therapy through corrected autologous tissues. We have formed a multidisciplinary team with the ultimate goal to develop an iPSC-based therapy for RDEB. Here, we present a clinical protocol that generates autologous, corrected epithelial keratinocyte sheets with the COL7A1 gene mutation corrected for grafting on to patients. We demonstrate the utility of sequential reprogramming and novel adenovirus-associated viral g

ORGANISM(S): Homo sapiens

SUBMITTER: Anthony Oro 

PROVIDER: E-GEOD-63101 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress