Emerging Gene Therapeutics for Epidermolysis Bullosa under Development.

ABSTRACT: The monogenetic disease epidermolysis bullosa (EB) is characterised by the formation of extended blisters and lesions on the patient's skin upon minimal mechanical stress. Causal for this severe condition are genetic mutations in genes, leading to the functional impairment, reduction, or absence of the encoded protein within the skin's basement membrane zone connecting the epidermis to the underlying dermis. The major burden of affected families justifies the development of long-lasting and curative therapies operating at the genomic level. The landscape of causal therapies for EB is steadily expanding due to recent breakthroughs in the gene therapy field, providing promising outcomes for patients suffering from this severe disease. Currently, two gene therapeutic approaches show promise f

SUBMITTER: Bischof J 

PROVIDER: S-EPMC10889532 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature