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Efficient CRISPR-Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome.


ABSTRACT: Exploiting the advantages of their highly efficient CRISPR-Cas9 gene editing strategy, in this study, Gálvez et al. abrogate SPINK5, the Netherton syndrome-causing gene, in primary human keratinocytes. SPINK5-knockout cells show disease hallmarks in vitro and in vivo, enabling the development of trustworthy models suitable for the evaluation of advanced therapies for genodermatoses.

SUBMITTER: Galvez V 

PROVIDER: S-EPMC7329935 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature

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