532 siRNA based non-viral gene therapy for the treatment of epidermolysis bullosa simplex(EBS)

EBS is an inherited, skin fragility disorder predominantly caused by dominant-negative mutations in genes encoding for the cytoskeletal proteins, Keratin5 and Keratin14 within the basal cell layer. To date, EBS is incurable with only symptomatic therapies currently available. The downregulation of these mutant genes would provide an excellent curative therapy for treating EBS. siRNA therapeutics have been identified as an attractive therapy option for EBS given the highly accessible nature of skin tissue.

Source: Journal of Investigative Dermatology - April 12, 2017 Category: Dermatology Authors: J. O'Keeffe Ahern, D. Zhou, L. Cutlar, I. Lara-S áez, W. Wang Tags: Genetic Disease, Gene Regulation and Gene Therapy Source Type: research