title: Stem Cell Therapy for Epidermolysis Bullosa-Does It Work?
abstract: Epidermolysis bullosa is a group of heritable skin fragility disorders with considerable morbidity and mortality. It is known to be caused by mutations in as many as 18 distinct genes, but there is no specific or effective treatment. Preclinical developments of gene correction, protein replacement, and cell-based approaches for treatment have suggested new therapeutic avenues, and some of them, including bone marrow transplantation and mesenchymal stem cell therapy, have entered into early clinical trials. Hammersen et al. report on two patients with severe generalized junctional epidermolysis bullosa treated with allogeneic stem cell therapy, but with little success. Careful examination of the existing literature suggests that current approaches of cell-based therapies may be helpful in ameliorating some of the clinical features and symptoms in these patients, but advanced strategies, with improved safety profiles, are required for development of durable therapy for these currently intractable disorders.
authors: Department of Stem Cell Therapy Science, Osaka University Graduate School of Medicine, Osaka, Japan.
authors: Katsuto
authors: K
authors: Tamai
authors: Department of Dermatology and Cutaneous Biology, Sidney Kimmel Medical College at Thomas Jefferson University, Philadelphia, Pennsylvania, USA; Jefferson Institute of Molecular Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania, USA. Electronic address: Jouni.Uitto@jefferson.edu.
authors: Jouni
authors: J
authors: Uitto
mesh_terms: D004820:Epidermolysis Bullosa / Q000235:genetics / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D033581:Stem Cell Transplantation / Q000379:methods*