Gene therapy to cure the Bubble boy disease
With technological advancement, medical science has progressed rapidly. Many innovations are taking place in the field of medicines and treatments. Doctors and scientists with rare conditions discover new ideas. Occurring of rare diseases or medical conditions make the researchers find a new treatment for the disease. It is studied that about a hundred babies are born with unique health conditions in the United States every year. The infants with this disease are born without an immune system. The immune system of these babies is not functional, which is very rare in any human. The condition called severe combined immune deficiency first occurred in 1984. A documentary Bubble Boy has made on a boy who has suffered from a similar rare disease in the mid-70s. But the movie was made on fiction then.
The boy who has suffered from this disease died in 1984 at the age of 12. The boy named David Vetter has spent most of his life inside a plastic bubble to prevent infection. Then he died at only 12 years of age. The babies born in the United States are now undergoing the test for this disease. The doctors recommend a bone marrow transplant to cure the disease. The operation is successful in about 90 percent of people. About 90 percent of the patients get a positive result through bone marrow transplant. The condition is becoming a matter of interest for many docto9rs and researchers. The doctors treated this disease in the 1990s in the European country. The process of the treatment involves removal of the infected blood cells and insertion of a new gene by the help of a virus. Then the cell is put back in the body, and the cells help in building the immune system in the patient’s body.
According to an immunologist in UCLA’s board center of regenerative medicine and stem cells research, Donald Kohn, the treatment through the gene insertion was successful in the 90s and earlier time of 2000s. With time, five of the patients who have undergone this process have developed leukemia. Eighteen of the patients who have gone through gene therapy are alive until now, but leukemia condition has made the scientists think further about gene therapy.
The scientists have worked on the gene therapy on how to insert a new gene in the body without the risk of leukemia. According to the latest report in the New England Journal of medicine, advanced gene therapy has been invented by scientists. In this process, the virus inserted to the patient in a modified HIV, which was engineered so that can’t be able to cause AIDS. The virus has reduced the risk of leukemia in the patient. The process is successfully incorporated in many patients over past decades, say the scientists of St. Jude Children’s Hospital in Memphis. The infants who have undergone this advanced gene therapy has developed healthy immune system now. The result of this treatment is outstanding according to the doctors who have treated the children with SCID.