Abstract
The use of direct gene transfer to correct genetic skin disease has been a longstanding goal in the gene therapy field. Here we demonstrate correction of the genetic disorder RDEB by the application of a replication defective herpes simplex virus-1 vector encoding the type VII collagen (C7) gene COL7A1, bercolagene telserpavec (BVEC), directly to skin via a phase 1-2 clinical trial. This trial was an intra-patient comparison of recurrent and chronic wounds administered with either topical BVEC or topical placebo applied every other day until healing.
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