New gene therapy capable of treating severe combined immunodeficiency – a serious disorder



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bubble boy disease

Some diseases called immunodeficiency diseases because of the fact that they cause a total destruction of the immune system. While some diseases are more critical than others, the "bubble boy" disease is a very dangerous disease that can affect the lives of children who suffer from it. In this illness, the patient may need to live in a sterile environment all his life, without being able to interact socially with people.

A recent study by a team of scientists at the St. Jude Children's Research Hospital has developed a new way to cure the disease. This is a major advance in the field of medicine, as people with this disease have rarely been able to undergo a bone marrow transplant that is not very effective and very dangerous.

The team was able to develop a treatment for this disorder called X-linked severe combined immunodeficiency (SCID-X1). The study and the results were published in the New England Journal of Medicine, where patients were able to produce T cells, B cells, and NK cells.

The study was led by Dr. Ewelina Mamcarz, a physician in St. Jude's bone marrow transplant and cell therapy department. He commented on the results: "These patients are now young children, who respond to vaccines and whose immune system makes all the immune cells they need to protect themselves from infections when they explore the immune system." world and lead a normal life. This is a first for patients with SCID-X1. "

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