The Bill & Melinda Gates foundation have awarded £2.3 million to the University of York, in attempt to push forward research into sickle cell anaemia
The project utilises a new technique developed by researchers at York and the Wellcome Sanger Institute to track and enumerate blood stem cells in people.
What is sickle cell anaemia?
Sickle cell anaemia is an inherited red blood cell disorder, those with the disorder do not have enough healthy red blood cells to carry oxygen throughout the body. It is the most serious type of sickle cell disease and is particularly common in people with an African or Caribbean heritage.
Around 100 million people have the sickle cell trait worldwide, but the trait itself does not cause the disease unless a child inherits the trait from both parents. This occurs much more frequently in areas where the trait is more common, sickle cell disease has a high mortality rate in children and is common throughout much of sub-Saharan Africa, affecting up to three percent of births in some parts of the continent.
The funding builds on earlier research done by the University of York which shows the power of an approach for identifying and monitoring potential cancer-causing mutations in gene therapy patients. The new two-year project from the University of York will see researchers extend the studies to a large cohort of patients in Africa to understand the potential risks of gene therapy.
Lead researcher Dr. Alyssa Cull from the York Biomedical Research Institute and Department of Biology said: “We are really excited by the opportunity to expand our studies to gather more robust real world data for leukaemia-related mutations in sickle cell patients.
“With so many institutions being involved in the research including new partners in Uganda and Tanzania, understanding the team will be able to understand the underlying risks of large-scale gene therapy and stem cell transplantation with respect to leukaemia development.”
Mutations
Dr Kent added: “Since gene therapies have been previously associated with the potential for initiating leukaemia (a blood cancer that can rapidly kill patients), tracking these mutations in patients over time becomes incredibly important for their future health.”
The team studied the genomes of single blood stem cells at extraordinary depths and were, able to show that sickle cell disease itself causes an increase in genomic damage. They then identified different mutations that occurred pre-and post-transplantation which can now be tracked in the future.
Research partners include the Wellcome Sanger Institute, Boston Children’s Hospital, MRC Uganda, and Muhimbili University of Health and Allied Sciences, Tanzania.
