591 A phase 1/2 study of genetically-corrected, collagen VII expressing autologous human dermal fibroblasts injected into the skin of patients with recessive dystrophic epidermolysis bullosa (RDEB)

We report the results of the ongoing Phase 1/2 clinical trial of an ex vivo gene therapy for the treatment of RDEB. Five adult subjects enrolled in this trial carried various null COL7A1 mutations resulting in undetectable C7 expression by immunofluorescence microscopy (IF) and a lack of intact anchoring fibrils (AF) by electron microscopy (EM).

Source: Journal of Investigative Dermatology - April 19, 2018 Category: Dermatology Authors: M. Marinkovich, A. Lane, K. Sridhar, D. Keene, A. Malyala, J. Maslowski Tags: Clinical Research: Patient Outcomes Research Source Type: research