"Groundbreaking Discovery: Scientists Unveil Game-Changing Mole Reversal Treatment for Rare Condition!"
Researchers at the Francis Crick Institute, UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital for Children (GOSH) have designed a new genetic therapy that could lighten debilitating giant moles in a rare skin condition.
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In the future, this treatment could potentially be used to remove giant moles, thereby reducing the risk of cancer in children and adults. It also has the potential to reverse other, more common types of risky moles as an alternative to surgery.
Small moles on the skin are common in society, but in congenital melanocytic nevus (CMN) syndrome, children are born with up to 80% of their bodies covered in large, painful, or itchy moles, which are caused by mutations acquired in the womb. These moles can sometimes develop into a serious type of cancer called melanoma.
Published in the Journal of Investigate Dermatology, the researchers silenced a gene called NRAS, which was mutated in these mole cells, in cells in the dish, and in mice. NRAS is included in a group of genes (RAS genes) which, when mutated, can cause moles and can predispose to cancer.
The team used a genetic therapy called RNA silencing, which silences mutated NRAS in mole skin cells. This therapy is delivered in special particles directly to the mole cells.
They gave mice with CMN an injection containing the therapy.
which reduced the shutdown of the NRAS gene in just 48 hours. They also tested it on cells and all parts of the skin of children with CMN. Silencing the gene triggers the mole cell to destroy itself.
Veronica Kinsler, Principal Group Leader of the Mosaicism and Precision Medicine Laboratory at the Crick, Professor of Pediatric Dermatology and Dermatogenetics at GOSH/UCL, and NIHR Research Professor, said, “CMN is a physical and mental challenge for children and adults living with this condition. and for their families.
“These results are very exciting, because the genetic therapy not only triggered the destruction of mole cells in the laboratory, but we also managed to insert them into the skin of mice.
These results suggest that future treatments could potentially reverse moles in humans. However, more testing is needed before we can give it to patients. “We are extremely grateful to our patients at Great Ormond Street Hospital, who have actively participated over many years to help us deliver this potential new therapy.
After further research, we hope this therapy can enter human clinical trials soon.”
Jodi Whitehouse, CEO of Caring Matters Now, which helped fund the research, said, “This breakthrough in finding a treatment for CMN could change the lives of the families we support living with CMN.
As someone who was born with CMN covering 70% of my body and has had 30+ surgeries in my childhood to try to remove CMN for fear of melanoma, to no avail, this news is very inspiring and encouraging.
This brings real hope to the lives of those living with CMN. "Catriona Crombie, Head of Rare Diseases at LifeArc, said, "This work is part of our commitment to improving the lives of people living with rare diseases.
by investing in promising research and helping scientists overcome barriers to translational research. If successful, we hope to see human clinical trials for this therapy in the next few years."
The researchers have worked closely with Crick's Translation team to develop the technology for the benefit of patients. This includes securing translational funding from LifeArc, to conduct more research in mice to understand how the treatment works over a longer period of time.
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