Leading edge: emerging drug, cell, and gene therapies for junctional epidermolysis bullosa.
Allison R KeithKirk TwaroskiChristen L EbensJakub TolarPublished in: Expert opinion on biological therapy (2020)
Emerging therapies address the cutaneous symptoms of JEB but are unable to attend to systemic manifestations of the disease. Investigations into the molecular mechanism(s) underpinning the failure of systemic allogeneic cell therapies are necessary to expand the range of effective JEB therapies.