Article
Antibody gene transfer treatment drastically improves epidermal pathology in a keratitis ichthyosis deafness syndrome model using male mice.
EBioMedicine - 1 Mar 2023
Peres Chiara, Sellitto Caterina, Nardin Chiara, Putti Sabrina, Orsini Tiziana, Di Pietro Chiara, Marazziti Daniela, Vitiello Adriana, Calistri Arianna, Rigamonti Mara, Scavizzi Ferdinando, Raspa Marcello, Zonta Francesco, Yang Guang, White Thomas W, Mammano Fabio
Abstract excerpt
BACKGROUND: Keratitis ichthyosis deafness (KID) syndrome is a rare disorder caused by hemichannel (HC) activating gain-of-function mutations in the GJB2 gene encoding connexin (Cx) 26, for which there is no cure, or current treatments based upon the mechanism of disease causation. METHODS: We applied Adeno Associated Virus (AAV) mediated mAb gene transfer (AAVmAb) to treat the epidermal features of KID syndrome...
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