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

doi:10.1016/j.stem.2026.01.001

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

Cell stem cell - 5 Feb 2026

Apaydin Dilem Ceren, Sadhnani Gaurav, Carlaw Tiffany, Renziehausen Jan, Lizunova Elena, Filor Viviane, Hiller Anna, Brumhard Sophia, Halim Vincent, Brüning Ulrike, Bischof Johannes, Marodin Rafaela Horbach, Kurek Daniel Z, Rhiel Manuel, Ammann Sandra, Cornu Tatjana I, Cathomen Toni, Sander Leif Erik, Obermayer Benedikt, Coscia Fabian, Kirwan Jennifer, Koller Ulrich, Gruber Achim D, Bäumer Wolfgang, Hedtrich Sarah

Abstract excerpt

Autosomal recessive congenital ichthyosis (ARCI) refers to a group of rare, highly debilitating skin disorders that significantly impair patients' quality of life and lack any effective treatment options. Here, we report clinically relevant in situ correction of the most common ARCI-causing mutation, TGM1 c.877-2A>G, a splice-site aberration, in human disease models. Targeted skin barrier modulation followed by...

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PMID: 41605220
DOI: 10.1016/j.stem.2026.01.001

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TGM1

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