Scientists from the University of British Columbia in Canada and the Berlin Institute of Health Research in Germany have collaborated to develop a new gene editing therapy that can be directly used to repair human skin - using CRISPR technology combined with nanoparticle "transport vehicles" to accurately correct pathogenic gene mutations. This achievement is expected to open up new treatment pathways for various genetic skin diseases, from rare diseases to common conditions such as eczema. The relevant research has been published in the latest issue of the journal Cell Stem Cell.
This therapy is aimed at autosomal recessive inherited ichthyosis (ARCI). This is a rare hereditary skin disease that appears at birth, with a incidence rate of about one in 100000. The patient's skin is extremely dry and scaly, accompanied by chronic inflammation and a high risk of infection. Currently, there is no cure and the patient relies on symptomatic treatment for life.
Research has shown that the new method can successfully repair the most common genetic defects that cause ARCI in models constructed from real human skin, and restore up to 30% of normal skin function. Previous studies have shown that this level is sufficient to achieve significant clinical improvements. More importantly, it directly addresses the root cause of the disease, and a single treatment may bring long-lasting therapeutic effects.
CRISPR and other gene editing technologies are bringing new hope to many diseases that were once considered 'unsolvable'. Despite rapid development, its application to the skin has long faced challenges: as the body's first barrier, the skin naturally blocks foreign substances, making it difficult for larger biological drugs to penetrate.
To this end, the team innovatively used lipid nanoparticles as "transport vehicles" to carry gene editing tools into cells. They first use clinically approved lasers to create painless micrometer sized pores on the surface of the skin, helping nanoparticles smoothly cross barriers and reach deep skin stem cells. Once in place, the gene editor accurately corrects erroneous DNA and awakens the skin's self repair ability.
The team stated that this platform technology has broad applicability and can quickly adapt to other genetic skin diseases.
