Researchers from the Francis Crick Institute, University College London (UCL) Great Ormond Street Institute for Child Health and Great Ormond Street Hospital for Children (GOSH) have created a new genetic therapy to alleviate debilitating moles in a rare skin condition.
Published in the Journal of Investigate Dermatology, the treatment could help prevent affected children and adults from developing cancer.
Covering up to 80% of children’s bodies at birth, congenital melanocytic naevus syndrome (CMN) is caused by progenitor-cell mutations during embryonic development that appear as large, painful or itchy moles, which can develop into severe melanoma.
Funded by the National Institute for Health and Care Research (NIHR), the Caring Matters Now Charity, the Patient Support Group, LifeArc and the NIHR Great Ormond Street Hospital Biomedical Research Group Centre, researchers used a genetic therapy known as silencing RNA, which works to block the action of the mutation NRAS, which is mutated in the cells in these moles, in cells in a dish and in mice.
When mutated, NRAS has the potential to cause normal cells to become cancerous.
After injecting the therapy into mice with CMN, researchers found the expression of the NRAS gene reduced after 48 hours.
Additionally, in cells and whole skin sections from children with CMN, silencing the gene triggered the mole cells to self-destruct.
Researchers aim to develop the technology for patient benefit, including translational funds provided by LifeArc, to carry out more research in mouse models to understand how the treatment works in the long term.
Veronica Kinsler, principal group leader, Mosaicism and Precision Medicine Laboratory, the Crick and professor of paediatric dermatology and dermatogenetics, GOSH/UCL, commented: “We have managed to deliver [the genetic therapy] into the skin in mice… [suggesting] that the treatment could potentially reverse moles in people.”
“After more studies in mice, we hope the therapy can soon enter clinical trials in people,” Kinsler added.

https://pharmatimes.com/news/researchers-design-new-genetic-mole-reversal-therapy-for-rare-skin-condition/