Malignant infantile osteopetrosis(MIOP) is a rare disease and in Sweden a child is born with MIOP one every three years, and world wide is one and every 300,000 births. Low bone density causes osteoporosis, but extremly high bone density is also harmful. High bone density is a rare, hereditary disease which can lead to children's death by age five. Researchers at Lund University in Sweden are now trying to delveop gene therapy against this disease. For the body to function there needs to be a balance between the cells that build up in the bones and the cells that break them down. MIOP, the cells break down the bone tissue do not function as they are suppose to causing the bone to have sufficient cavities for bone-marrow and nerves. Optic and auditory nerves are compressed and can cause blindness and deafness in children. If the bone marrow cease to functions, without treatment, the child could die of anemia and infections. Researchers are focuses on finding alternatives for treatments against MIOP, a bone-marrow transplant. This transplant is effective but can be risky and dependent on finding a suitable donor. Gene therapy requires no donor, stem cells are taken from the patients themselves. Once the cell's non functioning gene has been replaced with a healthy copy, the stem cells are placed back into the child. This method is used today for a blood disorder called thalassemia. Since it is impossible to tell control where the genes will end up this could cause leukemia. Gene therapy is only used for serious diseases because of the high risks. Lund researchers are trying to conduct this on plant cells and laboratory animals.
This is unfortante that children that have this conditon will be lucky if they reach age five. Although, researchers are trying to use gene therapy to try to replace the stems cells with their own healthy cells. This must be a diffucult decisions for parents to make wanting to help their child not suffer from opsteopetrosis but by using the gene therapy method could cause leukemia and now having another problem on their hands. I think reaserchers should come up with a safer procedure but since there is no cure for the diease besides possibly waiting on the donor list, I would still try the gene therapy procedure.
