Intravenous gentamicin therapy induces functional type VII collagen in patients with recessive dystrophic epidermolysis bullosa: an open-label clinical trial.
David T WoodleyMichelle HaoAndrew KwongBrandon LevianJon CoganYingping HouDaniel MosallaeiElana KleinmanKate ZhengClaire ChungGene KimDavid PengMei ChenPublished in: The British journal of dermatology (2024)
IV gentamicin induced the readthrough of nonsense mutations in patients with RDEB and restored functional C7 in their skin, enhanced wound healing and improved clinical parameters. IV gentamicin may be a safe, efficacious, low-cost and readily available treatment for this population of patients with RDEB.