First ever gene therapy gel corrects rare genetic skin condition

Peter Marinkovich MD, director of Stanford Medicine’s Blistering Disease Clinic

 

Stanford researchers have been working on gene therapies for epidermolysis bullosa, or “butterfly disease,” for over a decade. A new gel helped wounds heal and stay healed in a clinical trial … A gene therapy gel applied to the wounds of nine people — three of whom were children — with the blistering skin disease epidermolysis bullosa helped the wounds heal and remain healed for several months in a trial headed by researchers at Stanford Medicine … The trial is the first to show that gene therapy vectors for skin diseases can be effective when applied topically. It is also the first trial of gene therapy in children with epidermolysis bullosa … read more


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