842 Combining the reprogramming of RDEB fibroblasts with mono- and bi-allelic correction of the mutant COL7A1 gene into a one-step procedure

Coupling the reprogramming of somatic cells into induced pluripotent stem cells (iPSCs) with targeted gene correction using CRISPR/Cas9 offers the possibility of developing a new stem cell-based approach for the treatment of inherited skin blistering diseases such as Recessive Dystrophic Epidermolysis Bullosa (RDEB). The conventional iPSC-based therapy for RDEB involves multiple sequential steps such as reprogramming, gene correction and iPSC differentiation. Each of these steps complicates the manufacturing process and is associated with lengthy cell culture periods that increase the risk of mutation accumulation and karyotypic instability in the final epidermal cell product.
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research