Efficient CRISPR-Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome
Victoria Gálvez, Esteban Chacón-Solano, José Bonafont, Ángeles Mencía, Wei‐Li Di, Rodolfo Murillas, Sara Llames, Asunción Vicente, Marcela Del Río, Marta Carretero, Fernando Larcher
Abstract
. Our results demonstrate the feasibility of modeling genodermatoses, such as Netherton syndrome, by efficiently disrupting the causative gene to better understand its pathogenesis and to develop novel therapeutic approaches.
Topics & Concepts
BiologyGene knockoutCRISPRGenome editingTransgeneEx vivoCas9Genetic enhancementGeneGeneticsIn vivoSkin and Cellular Biology ResearchTransgenic Plants and ApplicationsSilk-based biomaterials and applications