Safety and wound outcomes following genetically corrected autologous epidermal grafts in patients with recessive dystrophic epidermolysis bullosa
Z Siprashvili, NT Nguyen, ES Gorell, K Loutit, P Khuu… - Jama, 2016 - jamanetwork.com
Importance Recessive dystrophic epidermolysis bullosa (RDEB) is a devastating, often fatal,
inherited blistering disorder caused by mutations in theCOL7A1gene encoding type VII
collagen. Support and palliation are the only current therapies. Objective To evaluate the
safety and wound outcomes following genetically corrected autologous epidermal grafts in
patients with RDEB. Design, Setting, and Participants Single-center phase 1 clinical trial
conducted in the United States of 4 patients with severe RDEB with a measured area of …
inherited blistering disorder caused by mutations in theCOL7A1gene encoding type VII
collagen. Support and palliation are the only current therapies. Objective To evaluate the
safety and wound outcomes following genetically corrected autologous epidermal grafts in
patients with RDEB. Design, Setting, and Participants Single-center phase 1 clinical trial
conducted in the United States of 4 patients with severe RDEB with a measured area of …