Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa
Arianna Tovar Vetencourt, Ibrahim O. Sayed-Ahmed, J. E. Gomez, Hubert Chen, Brittani Agostini, Kate Carroll, Trevor J. Parry, Suma Krishnan, Alfonso L. Sabater
Abstract
, which encodes type VII collagen. Blistering and scarring of the ocular surface develop, potentially leading to blindness. Beremagene geperpavec (B-VEC) is a replication-deficient herpes simplex virus type 1-based gene therapy engineered to deliver functional human type VII collagen. Here, we report the case of a patient with cicatrizing conjunctivitis in both eyes caused by dystrophic epidermolysis bullosa who received ophthalmic administration of B-VEC, which was associated with improved visual acuity after surgery.
Topics & Concepts
Epidermolysis bullosaMedicineDermatologyGenetic enhancementEpidermolysis bullosa simplexBlindnessVisual acuityPathologyOphthalmologyGeneBiologyGeneticsOptometrySkin and Cellular Biology ResearchSilk-based biomaterials and applicationsHair Growth and Disorders