903 Utilizing an organoid-based approach for the derivation of genetically corrected keratinocytes and fibroblasts in an induced pluripotent stem cell therapy for skin diseases

Induced pluripotent stem cells (iPSCs) hold promise for treating skin diseases such as epidermolysis bullosa (EB). An iPSC-based gene therapy EB involves reprogramming, correction, iPSC differentiation, and transplantation of genetically corrected iPSC-derived onto patients. One critical challenge this is inconsistent differentiation iPSCs into high-quality keratinocytes (iKCs) fibroblasts (iFBs). To address challenge, we derive iKCs iFBs from human organoids, which successfully generate by adapting a protocol published Lee et al. date, have generated 4 lines using our patented combined editing reprogramming approach. We also differentiated these modified organoid-based procedure. Importantly, the resulting engrafted immunocompromised mice formed functional dermis epidermis. discovered that co-delivery wound area improves formation. These results are consistent with previous observation both needed formation basal membrane in Our success production organoids will now simplify reduce cost manufacturing cell since all necessary types produced one