Molecular therapy : the journal of the American Society of Gene Therapy
Base edited skin equivalents with clinical potential for ex vivo correction of recessive dystrophic epidermolysis bullosa.
Alex Bassons-Bascuñana, Blanca Duarte, Estela Méndez-Jiménez, Laura Ugalde, Paula Sanabria-Betrián, Clotilde Aussel, Irene Peña-Gutiérrez, Beatriz Olalla-Sastre, Sandra Ammann, Toni Cathomen, Marcela Del Rio, Paula Río, Fernando Larcher, Sergio López-Manzaneda
Published: 202510.1016/j.ymthe.2025.11.023
Abstract
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…
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