Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa.
N Engl J Med
; 390(6): 530-535, 2024 Feb 08.
Article
em En
| MEDLINE
| ID: mdl-38324486
ABSTRACT
Dystrophic epidermolysis bullosa is a rare genetic disease caused by damaging variants in COL7A1, 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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Epidermólise Bolhosa Distrófica
/
Colágeno Tipo VII
Tipo de estudo:
Etiology_studies
Limite:
Humans
Idioma:
En
Revista:
N Engl J Med
Ano de publicação:
2024
Tipo de documento:
Article