New England Journal of Medicine · 2024 · 13 citations · 7 references
MedicineOphthalmologySurgeryGene TherapyOcular DiseaseOphthalmic SurgeryConjunctival DiseaseConjunctival PathologyOcular Gene DeliveryOcular Gene TherapyOcular Surface DiseaseDystrophic Epidermolysis BullosaEpidermolysis Bullosa SimplexEpidermolysis Bullosa SyndromesOphthalmic AdministrationVisual Acuity ImpairmentVisual Acuity Improvement
, 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.
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In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial
Irina Gurevich, Pooja Agarwal, Peipei Zhang et al. · Nature Medicine · 2022 · 161 citations · Full text
Medicine, Dermatology, Pathology +10