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

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

doi:10.1016/j.stem.2026.01.001

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.

No abstract yet

We only have this paper's title and publication details.

Read it at the source

Ask about this paper

Answers are grounded in this paper and cite their sources.

Implication

Observational analysis reveals successful gene editing of congenital ichthyosis mutations in human skin models, suggesting a novel gene therapy approach.

Discussion

No takes yet. Share an insight, caveat, or question.

Cite This Study

Apaydin et al. (2026) studied this question.

synapsesocial.com/papers/69a75a57c6e9836116a200cb https://doi.org/10.1016/j.stem.2026.01.001

View at Publisher

Ask AI

Bookmark

Share

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

Key Points

No abstract yet

Ask about this paper

Implication

Discussion

Cite This Study

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

Key Points

No abstract yet

Ask about this paper

Implication

Discussion

Cite This Study

Keep reading

Keep reading