Article
Base edited skin equivalents with clinical potential for ex vivo correction of recessive dystrophic epidermolysis bullosa.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Apr 2026
Bassons-Bascuñana Alex, Duarte Blanca, Méndez-Jiménez Estela, Ugalde Laura, Sanabria-Betrián Paula, Aussel Clotilde, Peña-Gutiérrez Irene, Olalla-Sastre Beatriz, Ammann Sandra, Cathomen Toni, Del Rio Marcela, Río Paula, Larcher Fernando, López-Manzaneda Sergio
Abstract excerpt
Recessive dystrophic epidermolysis bullosa (RDEB) is a severe genetic disorder caused by bi-allelic null mutations in the COL7A1 gene. Among various therapeutic genome editing (GE) strategies, adenine base editors (ABEs) have demonstrated particular efficacy in nonsense mutations. Previous studies have predominantly focused on RDEB fibroblasts, which produce significantly less type VII collagen (C7) than...
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