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A genetic immunodeficiency disorder is catching the attention of the public. However, an iconic John Travolta features a film on “bubble boy” disease. This disease is known as severe combined immunodeficiency disease (SCID).
People who are suffering from this disease there is an essential update for them. After decades, researchers are showing a study on a big screen from St. Jude Hospital and accepted a Smithsonian Ingenuity Award for what is referred for the cure.
SCID is a disease, and less than 20 infants affect with it every year. It is due to mutations in the gene that develop in the immune system. People born with SCID especially males are unable to fight against infections.
Other dynamic viruses like pneumonia can cause death in many SCID patients before age one.
What Does happen In this Method?
In the first treatment level, individuals put in NASA level protective suits for insulating them from germs. This method is so difficult to live a healthy life.
As you know in hospitals, bone marrow transplants method is used to treat this condition. But for that, you need a sibling who could match.
A physician and survivor of childhood cancer patient determined to save the kids with SCID. The physician name is Sorrentino, and he finds a way to initiate the growth of an immune system in newborn.
According to his research, the use of a virus to transport and insert a copy of a gene into the genome of patients, ” blood stem cells.” St Jude also Funded the research along with the California Institute of Regenerative Medicine and the National Institutes of Health.
A researcher of St. Jude, Ewelina Mamcarz told to Yahoo Lifestyle in this method taking a deactivated HIV and using it to deliver an unmutated gene. Moreover, Dr Sorrentino said, what are viruses good at? they are good at infecting cells.
Mamcarz who passed away due to lung cancer last year said to the doctor he used the property of that virus to carry the normal growth of the gene into the cell.
According to Mamcarz, Almost 12 infants with SCID have undergone. Firstly bone marrow cells remove from the patient and then expose them to the deactivated HIV that contains the healthy gene for three days.
After that, infected cells cryopreserved for two weeks; meanwhile, a patient undergoes a two-day treatment of chemotherapy to make a place for new cells.
So when they are ready, the cells delivered back to infant through two to three-minute infusion. After that, immune systems start to develop.
Mamcarz said, these are smart cells and know very well where to go.?
All 12 patients who received the treatment started to develop a healthy and strong immune system and live a normal life.
Furthermore, Marcarz tells Yahoo Lifestyle, ” there is no need for any protective isolation or medications anymore. The first trial of this therapy is spending a healthy life, and three years duration have passed. They enjoy life, go on a family trip and no different than other kids.
The treatment is in Phase I clinical trial and focuses on efficacy and safety. Well, the team of researchers performed very well and now partners at the University of San Francisco and the National Institutes of Health.