Scientists Bobby Gaspar and Adrian Thrasher together with Dr Marlene Carmo are making great strides in the Development of Gene Therapy for Familial HLH which is being funded by the H R Trust at the Institute of Child Health, London.
The team are looking at replacing Perforin, the commonest gene to cause inherited HLH. They have discovered that in order to cure HLH, patients who have an abnormal gene need to be given a normal perform gene. To achieve this, viruses called lentiviruses are used to carry the normal gene into the patients’ cells. To read more, look under our Latest Research section.