Gene-editing treatment for skin conditions developed at UBC

A University of British Columbia team reported a topical gene-editing therapy that corrected faulty genes in human skin models and restored up to 30% of normal function for a rare inherited disorder, in results published in Cell Stem Cell.

Researchers at the University of British Columbia, together with collaborators in Germany, report a topical gene therapy that can correct faulty genes in human skin models. The work appears in Cell Stem Cell and targets genetic skin disorders that currently lack effective cures. The team tested the approach on models of autosomal recessive congenital ichthyosis (ARCI) and measured partial restoration of skin function. Their delivery method uses lipid nanoparticles and a clinically approved laser to reach skin stem cells beneath the surface. Key facts: - The study is reported by UBC in collaboration with the Berlin Institute of Health at Charité and published in Cell Stem Cell. - In living human skin models, the treatment corrected the most common mutation behind ARCI and restored up to 30% of normal skin function. - Delivery combines lipid nanoparticle (LNP) technology with microscopic, pain-free openings created by a clinically approved laser to allow the therapy to reach skin stem cells. - The researchers reported the approach is localized to the skin and saw no evidence of off-target effects in their tests. - The project involved NanoVation Therapeutics, a Vancouver biotech spin-off focused on LNP-based genetic medicines. - The team is working with regulatory authorities and aims to move toward first-in-human clinical trials. Summary: The reported method introduces a localized, topical route to apply gene editing directly to human skin and is presented as a platform that could be adapted for multiple genetic skin conditions. The team is engaging in regulatory discussions and planning for first-in-human clinical trials to evaluate safety and efficacy.