Editing the skin in place: In vivo genome correction of rare skin disease.
editorial · Level V
Where this comes from
- Record sourced from PubMed, PMID 41650933.
- Also identified by DOI 10.1016/j.stem.2026.01.003.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
Abstract
In this issue, Apaydin and Sadhnani et al. report in situ genome editing of human skin to correct a common disease-causing mutation underlying autosomal recessive congenital ichthyosis (ARCI).<sup>1</sup> They combine a base editor, transient barrier modulation, and topical mRNA-lipid nanoparticle administration to restore clinically meaningful levels of transglutaminase 1 activity.
Medical subject headings
- Gene Editing
- Skin
- Skin Diseases