Evaluation of prenatal intra-amniotic LAMB3 gene delivery by adenoviral and AAV vectors in a mouse model of Herlitz junctional epidermolysis bullosa

Background: Prenatal gene therapy has been considered for diseases such as Herlitz junctional epidermolysis bullosa (H-JEB), a lethal genodermatosis caused by absence of any of the three subunits of the basement membrane protein laminin-5. H-JEB presents from birth with widespread blistering of skin and mucosae and leads to death typically within the first year of life. In this study, we investigated in a mouse model whether H-JEB could be treated by gene delivery in utero. Methods: Adenovirus type 5- and AAV2-derived vectors carrying a reporter gene or LAMB3 cDNA encoding the beta3 chain of laminin-5 were gener…