131 Phase I/II efficacy and safety study of mesenchymal stromal cells in recessive dystrophic epidermolysis bullosa

Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) have a pronounced muco-cutaneous fragility, which triggers the formation of blisters spontaneously or in response to minimal trauma. RDEB is also associated with chronic inflammation; patients have wounds that do not close and aggressive squamous cell carcinomas. The disease is due to mutations in the COL7A1 gene that codifies for collagen VII (C7). One of the most promising therapeutic options is the use of mesenchymal stromal cells (MSCs) from healthy donors that, in addition to producing C7 and having anti-inflammatory properties, are well tolerated by the patient ’s immune system.
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Clinical Research and Epidemiology Source Type: research