Active Pregnancy, Children & Inherited Conditions NIHR-supported project Digestion, Kidneys & Other Organs

Topically Gene therapy for genetic skin condition Ichthyoses

In plain English

AI plain-English summary

A gel smeared onto the skin could correct the faulty genes that cause severe, lifelong skin disorders. Congenital ichthyoses are genetic conditions that leave patients with painfully thick, red, and itchy skin covering their entire bodies, often requiring disability support, and no curative treatments exist. The researchers have already developed a gene-editing therapy that corrects the underlying genetic error with high efficiency in the lab. Their new approach packages the editing tools into harmless, virus-like particles mixed into a gel, which can be applied directly to the skin rather than injected. If this works in patients, it would be the first curative treatment for ichthyosis, replacing a lifetime of symptom management with a single topical application. The gel-based delivery system could also be adapted for other genetic skin conditions, potentially transforming how fundamental gene-editing technology reaches patients in a practical, non-invasive way.

View original technical description
ICH Lab Study Congenital ichthyoses (CI) are a group of genetic skin disorders with symptoms of severe dryness, skin thickening, itching and redness. They can be debilitating, significantly impacting quality of life with many of those affected needing disability support throughout their lives. There are no curative treatments. We have been developing gene editing therapy for this condition by correcting the faulty gene using gene editing technology and have achieved high correction efficiencies. Since ichthyosis patients have skin lesions all over the body, we propose to directly deliver gene editing reagents to the skin by using harmless engineered virus-like particles in gel formation.

Researchers

Wei-Li Di (Principal Investigator)

Related Research

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Leveraging genetic models of skin barrier dysfunction to gain insights into cutaneous inflammation
Identification of novel targetable signalling pathways in the inherited ichthyoses
Understanding the dysfunctional skin barrier in severe ichthyosis.
Understanding the dysfunctional skin barrier in severe ichthyosis
Gene therapy for rare genetic disease Keratitis-Ichthyosis-Deafness Syndrome - Correction of Faults of the Disease-Associated Gene GJB2 by Prime Gene

Original classification

Gene, stem and cellular therapies

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