Current Research
Gene therapy is currently being further researched and improved. Gene therapy trials for SCID were once halted worldwide for a number of years when it was reported that children who had been treated for XSCID in a French gene-therapy experiment had developed a type of leukemia. They discovered that the mechanism used to insert the corrective gene had placed it in a region of a receiving cell's chromosome which switched on a cancer-causing gene called an oncogene. Today, NHGRI ( National Human Genome Research Institute) researchers are evaluating insertion profiles and gene delivery methods. The goal of this work is to find ways to achieve permanent correction of the DNA while avoiding the activation of oncogenes.
NHGRI investigators are also studying to improve the effectiveness of bone marrow transplantation. A major limitation to bone marrow transplantation is that blood-forming stem cells are not well understood. They are few in number and cannot be purified or expanded outside the body for very long. NHGRI researchers are investigating the roles of genes that control the reproduction and differentiation of blood-forming stem cells which increase the supplies of these life-saving cells for clinical use and patients of SCID.
This current research is just one of many. Research is such a vital necessity of improving and sustaining life of children with genetic diseases. Without research, numerous treatments and tests that cure so many patients today would never have been discovered.
Gene therapy is currently being further researched and improved. Gene therapy trials for SCID were once halted worldwide for a number of years when it was reported that children who had been treated for XSCID in a French gene-therapy experiment had developed a type of leukemia. They discovered that the mechanism used to insert the corrective gene had placed it in a region of a receiving cell's chromosome which switched on a cancer-causing gene called an oncogene. Today, NHGRI ( National Human Genome Research Institute) researchers are evaluating insertion profiles and gene delivery methods. The goal of this work is to find ways to achieve permanent correction of the DNA while avoiding the activation of oncogenes.
NHGRI investigators are also studying to improve the effectiveness of bone marrow transplantation. A major limitation to bone marrow transplantation is that blood-forming stem cells are not well understood. They are few in number and cannot be purified or expanded outside the body for very long. NHGRI researchers are investigating the roles of genes that control the reproduction and differentiation of blood-forming stem cells which increase the supplies of these life-saving cells for clinical use and patients of SCID.
This current research is just one of many. Research is such a vital necessity of improving and sustaining life of children with genetic diseases. Without research, numerous treatments and tests that cure so many patients today would never have been discovered.