Highlights of Gene and Cell Therapy for Epidermolysis Bullosa and Ichthyosis
August 2024
in “
Dermatology and Therapy
”
TLDR Gene and cell therapies are being developed to treat rare skin conditions like epidermolysis bullosa and ichthyosis.
Advancements in molecular genetics have identified genetic variants causing epidermolysis bullosa (EB) and ichthyosis, leading to the development of gene and cell therapies for these rare skin conditions. Despite limited application to rare inherited skin conditions, successful examples of in vivo gene therapy and ex vivo cell therapy have been developed for EB and ichthyosis. The review highlights preclinical research and clinical development, including a gene therapy recently approved by the U.S. FDA for recessive dystrophic EB.