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Lipid nanoparticle-based non-viral in situ gene editing of congenital ichthyosis-causing mutations in human skin models | Synapse
March 3, 2026
Lipid nanoparticle-based non-viral in situ gene editing of congenital ichthyosis-causing mutations in human skin models
DA
Dilem Ceren Apaydin
GS
Gaurav Sadhnani
TC
Tiffany Carlaw
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Key Points
Successful gene editing resulted in corrected mutations associated with congenital ichthyosis, improving skin morphology.
The approach utilized lipid nanoparticles to deliver gene editing components effectively into targeted skin cells.
Assessment of outcomes was conducted through in situ gene editing in human skin models, ensuring direct relevance to human health.
This innovative method may enable future therapeutic strategies for congenital skin disorders, pending further validation.
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Apaydin et al. (Tue,) studied this question.
synapsesocial.com/papers/69a75a57c6e9836116a200cb
https://doi.org/https://doi.org/10.1016/j.stem.2026.01.001
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