Article
Emerging Gene Therapeutics for Epidermolysis Bullosa under Development.
International journal of molecular sciences - 13 Feb 2024
Bischof Johannes, Hierl Markus, Koller Ulrich
Abstract excerpt
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...
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