Researchers Use Gene Therapy To Build Immune Systems For Patients With Bubble-Boy Disease

Researchers from St. Jude Children’s Hospital might have just developed a new gene therapy that can build a healthy, functioning immune system for patients suffering from severe combined immunodeficiency, SCID, reported Cincinnati, a sector of the USA TODAY Network. For children with this disease, this could be a huge breakthrough and potential permanent cure. These patients often don’t live passed the age of two-years-old, but now these researchers might have exactly what they’ve been needing.

Severe combined immunodeficiency (SCID), more popularly known as “bubble-boy disease” is a disease in which one’s immune system fails, and every day activities pose a threat to the patient’s life. This occurs when a child is born without the ability to produce healthy T & B-cells, as well as natural cell killers that protect the body from infection. These cells are the warriors of the immune system. The disorder is onset from a mutated protein gene, that affects mostly just males. About 1 in 50,000-100,000 babies experience this gene deformity.
The study was led by Dr. Brian Sorrentino, along with Dr. Ewelina Mamcarz. These two are very pleased with the work they have found, especially since it’s the first time ever that a clinical trial developed healthy immune systems for patients with SCID. Just four months after the trial, their bodies were developing normal antibodies on their own, something they were unable to do before.

The current procedure to treat the disease is by performing a bone marrow transplant, yet it’s common that a perfect match doesn’t exist for the patient. Nearly 80% of patients face risks. While the transplant has been successful for a select few, the new gene therapy is hoping the treatment that will surpass the current and positively affect all those diagnosed.

While the St. Jude researchers are confident in the future of their gene therapy treatment, they is still a ways to go from here. They will need to track these patients’ future progress to see if the “new” immune system holds up and builds its strength.

Not only is this exciting news for the SCID community, but researchers are hopeful that gene therapy will have positive affects on other diseases as well.


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