[HTML][HTML] Phase I/II open-label trial of intravenous allogeneic mesenchymal stromal cell therapy in adults with recessive dystrophic epidermolysis bullosa

E Rashidghamat, T Kadiyirire, S Ayis, G Petrof… - Journal of the American …, 2020 - Elsevier
Background Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary blistering
disorder due to a lack of type VII collagen. At present, treatment is mainly supportive.
Objectives To determine whether intravenous allogeneic bone marrow–derived
mesenchymal stromal/stem cells (BM-MSCs) are safe in RDEB adults and if the cells
improve wound healing and quality of life. Methods We conducted a prospective, phase I/II,
open-label study recruiting 10 RDEB adults to receive 2 intravenous infusions of BM-MSCs …