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Lipid nanoparticle-based non-viral in situ gene editing of congenital ichthyosis-causing mutations in human skin

Dilem Ceren Apaydin1, Gaurav Sadhnani1, Tiffany Carlaw2

  • 1Berlin Institute of Health, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt Universität zu Berlin, Berlin 10117, Germany.

Cell Stem Cell
|January 28, 2026
PubMed

Keywords:
ARCIautosomal recessive congenital ichthyosiscytosine base editorfunctional rescuegene therapygenomic safetyin situ gene editingin vivo gene therapylipid nanoparticlestransglutaminase 1

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