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Mice study offers new hope for skin disease

Ada and family

Research from the UK using genetic therapy and mice studies has uncovered a potential treatment to reverse a rare skin disease.

Congenital melanocytic naevus syndrome (CMN) is a rare skin condition that creates giant moles which can lead to skin cancer (melanoma).

The study, at the Francis Crick Institute, London, developed a genetic therapy that can counteract the effect of an inherited mutated gene (named NRAS) which is directly responsible for this skin condition.

Veronica Kinsler, lead researcher, said, "These results are very exciting, as not only does the genetic therapy trigger self-destruction of the mole cells in the lab, but we have managed to deliver it into the skin in mice."

Researchers used RNA molecules, enclosed in particles, to target the mutated gene in genetically altered mice with CMN. This then led to the reduced production of the NRAS gene.

Ada, a 3-year-old girl with 70% of her body covered in CMN (pictured centre with her parents, Rachelle and Greg), contributed skin cells for the research.

Her parents said: "Knowing there has been a huge step forward in CMN research and there could be a chance of Ada’s CMN being reversed and possibly reducing Ada’s risk of developing melanoma, has blown our expectations out of the water."

This research – published in the Journal of Investigative Dermatology – promises safer, more effective treatments for CMN, with future studies focusing on long-term effects in animals and potential applications in humans.


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