The impact our Research Training Fellows make is great. Their work had led to patient and healthcare sector benefits:
- Influencing policy and guidelines
- Improving clinical practice and patient care on topics such as vaccine use, epilepsy, pregnancy management, preterm birth and developing diagnostic techniques
- Developing treatments for life-threatening and life-limiting conditions.
Bobby Gaspar was awarded an Action fellowship back in the 1990s. He studied two life-threatening immunodeficiency disorders, which occur when the body’s natural immune system is absent or not working properly. At this time the conventional treatment for these disorders was bone marrow transplant, which carries risk if the donor match is poor. Professor Gaspar’s pioneering work has led to new gene therapy treatments which are now saving children’s lives.
Professor Gaspar comments: “I received three year Research Training Fellowship funding which allowed me to complete my PhD. This was my academic foundation, it set me on the road to my academic and clinical career.”
Around 25,000 children in the UK are blind or partially sighted. It is often unclear exactly what causes these children’s eyesight problems, making it difficult to identify the best treatment. Thanks to Dr Lily Islam, with a Research Training Fellowship from Action, a number of genetic changes that cause childhood blindness have been discovered and a new test developed to help explain the causes of specific eyesight problems.
Dr Islam says: “In my experience, families greatly value having a genetic diagnosis. It means we can explain why their child has eyesight problems and how their vision might change in the future. We can also offer families genetic counselling, if they want to find out what their chances are of having another baby affected by eyesight problems.”