Congenital melanocytic nevus syndrome can cover up to 80% of a child’s body.
Researchers from the Francis Crick Institute, UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Sick Children (GOSH) have developed a new gene therapy to reduce debilitating moles caused by a rare skin condition.
Published in Journal of Dermatology ResearchThis treatment could help prevent the development of cancer in affected children and adults.
Congenital melanocytic nevus syndrome (CMN), which covers up to 80% of a child’s body at birth, is caused by mutations in precursor cells during embryonic development and appears as a large, painful, itchy mole that can develop into a severe melanoma.
Funded by the National Institute for Health and Care Research (NIHR), Caring Matters Now Charity, patient support groups, LifeArc and the NIHR Great Ormond Street Hospital Biomedical Research Group Centre, the researchers used a gene therapy called silencing RNA, which works to block the action of mutant NRAS in the cells of these moles, in cells in culture dishes and in mice.
When NRAS is mutated, it can cause normal cells to become cancerous.
The researchers found that after injecting the treatment into CMN-treated mice, 48 hours later, expression of the NRAS gene was reduced.
They further found that silencing the gene caused mole cells to self-destruct in cells and whole skin of children with CMN.
With the help of translational funding provided by LifeArc, the researchers aim to develop the technology to benefit patients and conduct further studies in mouse models to understand how the treatment works in the long term.
Veronica Kinsler, lead group leader at the Crick Institute for Mosaic and Precision Medicine and professor of paediatric dermatology and dermatogenetics at GOSH/UCL, said: “We were able to successfully deliver[the gene therapy]to the skin of mice…[this]suggests that it may be possible to reverse moles in humans with this treatment.”
“After further studies in mice, we hope this treatment will soon enter human clinical trials,” Kinsler added.
