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AIDS Virus Used In Gene Therapy To Treat The Baby Bubble Disease

AIDS virus is the short form of Acquired Immunodeficiency Syndrome, and it is also referred to as Human Immunodeficiency Virus (HIV). HIV is a condition where the immune system gradually fails and leads to the causation of various infections, which finally leads to death. Without treatment, it is observed that the average survival rates of HIV patients will be 9 to 11 years. With advanced research and technology, scientists and doctors have found a cure for HIV. HIV infects vital cells in the human immune system, such as helper T cells, macrophages, and dendritic cells. The HIV infection leads to lower helper T cells, leading to direct killing of the cells and weakening the immune system.

AIDS Virus Used In Gene Therapy To Treat The Baby Bubble Disease

Gene therapy is a technique used by doctors and scientists to use the gene to treat and prevent a particular disease. This technique does not use surgery. Instead, the doctors treat it by inserting the gene. The disease bubble baby is also known as severe combined immunodeficiency (SCID), which a very rare disease caused by distributed development of functional cells T cells & B cells which are caused by numerous mutations which can result in different clinical presentations.

AIDS Virus Used In Gene Therapy To Treat The Baby Bubble Disease

Research shows that the AIDS virus can be used used in gene therapy to make a working immune system by replacing the genes babies born without it. On Tuesday, the news reported that the unlikely helper AIDS virus gave a working immune system to 48 babies and toddlers who were born without one. Results also show that more than 50 babies and toddlers now have the germ-fighting ability because of gene therapy.

The study leader of UCLA Mattel Children’s Hospital reported that they are taking what otherwise would have been a fatal disease and the children are healing in the first dose of treatment to do the normal things like going to school, playing without worrying about the causing infections. The other two children were not cured with gene therapy, but they were cured with the bone marrow transplant. However, to know whether the disease has been cured or not might take a much longer time, but as of now, all seem well.

The children with Severe Combined Immunodeficiency Syndrome are caused by an inherited genetic flaw that keeps the bone marrow from making a healthy version of blood cells that form the immune system, which can make the baby die within a year or two. This disease is also known as bubble baby disease because it was found in 1970 in Texas. A boy lived for 12 years in a plastic bubble to be isolated and protect himself from germs and infections. More than 20 different types of gene defects can cause to girls or boys, or both. Bone marrow transplant from a suitable donor can cure the disease, but it is difficult to find a suitable donor, and it is a risky transplant. The 12-year-old boy died after his first bone marrow transplant.

Now the patients have been treated with antibiotics and germ-fighting antibodies weekly twice, but the doctors think it’s not a permanent solution. But the gene therapy might be, what they do is they remove some of the blood cells from the patient and use the disabled AIDS virus and insert int into the patient’s gene and then it is inserted back into the patient with the help of IV.

The case of an 11-year-old Josselyn Kish, who now lives in Los Vegas, had SCID when she was three years old. Before the gene therapy, Josselyn used to have rashes, painful shingles, and frequent diarrhea, and her preschool teacher used to call frequently as she used to have a fever all the time. But now, after the gene therapy, she hardly falls sick and she is stronger than ever, her mother Kim Cater mentioned in one of the reports. And recently, she was diagnosed with covid-19 and she is showing mild symptoms.

It is always asked that is gene therapy is a permanent cure? But who knows, maybe in the long term, but for now, it is working and the children are doing well.

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