X-linked lymphoproliferative disease
Developing a new treatment to cure boys with XLP
X-linked lymphoproliferative disease (XLP) is a rare but severe, inherited disorder of the immune system, caused by a faulty gene, that affects an estimated 300 to 500 boys worldwide.
The most common effect of XLP is that the immune system over-reacts to certain viral infections with serious, sometimes life-threatening, complications. In addition, about a third of patients develop lymphoma, a cancer of the immune cells.
The only available treatment is a bone marrow transplant, but the results depend on having a well-matched donor at the right time. If transplants come too late or donors can’t be found, boys are at risk of losing their lives.
That's why we're funding research, led by Professor Claire Booth at UCL Great Ormond Street Institute of Child Health, that aims to use new gene editing technologies to develop a cure for XLP that doesn't involve waiting for a bone marrow donor.
If our results are promising, this could pave the way to a clinical trial and lead to an exciting new treatment for boys with XLP.
Professor Booth's project builds on previous work by the team that has shown gene-edited white blood cells (T-cells) can correct some symptoms of XLP in the laboratory. They now want to use this same approach and recent advances in gene editing technologies to safely correct the faulty gene that causes XLP in a child’s own blood stem cells.
If successful, this work could correct all cells of the immune system – completely curing boys of this devastating condition and it could also accelerate progress towards similar cures for other genetic diseases – saving even more lives in the future.
It’s exciting and a great privilege to work with Action who are championing research in a range of diseases, like XLP, so that we can work towards safer treatments and cures.
Together we can fight XLP
100% of your donation will support groundbreaking research that offers hope of curing XLP