Scientists develop groundbreaking gene therapy to fix skin disorders

Scientists develop groundbreaking gene therapy to fix skin disorders

In a world-first breakthrough, researchers at the University of British Columbia (UBC) have developed a topical gene therapy capable of correcting faulty genes in human skin, offering hope for permanent cures for a wide range of genetic and common skin conditions.

Working alongside the Berlin Institute of Health at Charité, Germany, the team successfully applied this therapy to living human skin models. The study, published in Cell Stem Cell, demonstrates that the treatment can restore up to 30 percent of normal skin function—a clinically significant improvement that could transform patient care.

Treating the root cause, not just symptoms

Dr. Sarah Hedtrich, associate professor at UBC’s School of Biomedical Engineering, explained that the therapy directly targets the underlying genetic mutations causing disease.

“This treatment is safe, scalable, and easy to use,” said Dr. Hedtrich. “Our data suggests a single application could offer a long-lasting cure, treating the root cause rather than just alleviating symptoms.”

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The therapy has shown success in autosomal recessive congenital ichthyosis (ARCI), a rare but severe condition causing extremely dry, scaly skin, chronic inflammation, and high risk of infections. Currently, ARCI patients must manage symptoms throughout life with no effective treatment.

A versatile platform with broad potential

While ARCI is rare, this therapy could be adapted to treat other genetic skin disorders, including epidermolysis bullosa (“butterfly skin”), and potentially more common conditions such as eczema and psoriasis.

“This is a versatile platform,” Dr. Hedtrich added. “It can be adapted for almost any skin disease, opening new horizons for dermatology and personalized medicine.”

Innovative delivery overcomes skin barriers

The skin’s protective role has long made gene therapy delivery a challenge. The team overcame this by combining microscopic, painless laser openings with lipid nanoparticles, a safe delivery method also used in mRNA vaccines.

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These nanoparticles carry the gene-editing therapy into skin stem cells, where it corrects the faulty DNA and restores normal skin function. Tests confirmed no off-target effects, a critical milestone in safety.

Next steps: Clinical trials

The study was conducted in collaboration with NanoVation Therapeutics, a UBC spin-off focused on LNP-based genetic medicines. Researchers are now preparing for first-in-human trials, working closely with regulatory authorities to ensure safety and efficacy.

“Our goal is to bring a safe, effective treatment to patients who currently have no real options,” Dr. Hedtrich said. “This therapy could truly transform lives.”

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