
Dr. Sarah Hedtrich (center) and her team examine a skin-on-a-chip model used to test the new CRISPR-based therapy on living human skin samples.
Photo Credit: UBC Faculty of Medicine.
Scientific Frontline: "At a Glance" Summary
- Main Discovery: Researchers developed the first topical CRISPR-based gene therapy capable of correcting disease-causing mutations directly within human skin tissue.
- Methodology: The treatment utilizes lipid nanoparticles (LNPs) to deliver gene-editing machinery into skin stem cells through microscopic, pain-free channels created by a clinically approved laser.
- Key Data: In living human skin models of autosomal recessive congenital ichthyosis (ARCI), the therapy restored up to 30 percent of normal skin function, a level considered clinically meaningful.
- Significance: This breakthrough overcomes the skin's protective barrier to enable localized, potentially permanent genetic correction without the safety risks of systemic off-target effects.
- Future Application: The platform is being adapted for other severe genetic skin diseases like epidermolysis bullosa, as well as common conditions like eczema and psoriasis, with plans for first-in-human clinical trials.
- Branch of Science: Biomedical Engineering, Dermatological Genetics, and Nanomedicine.

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