Gene therapy is safe and can help improve the immune system over time, according to the National Institute of Allergy and Infectious Disease.
Scientists from the National Institute of Allergy and Infectious Disease have found that gene therapy is safe for older children and young adults who are suffering from a rare inherited disorder, X-linked severe combined immunodeficiency (SCID-X1).
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SCID-X1 is mostly caused by the mutation in IL2RG and primarily affects males. The mutation prevents infection-fighting immune cells for developing properly and hinders immune system’s ability to fight infectious diseases. A person born with SCID-XI is at high risk of catching life-threatening infections and may lead to severe illness and even to death.
Stem cell transplant is the most effective treatment for this disorder so far in which the defective bone marrow is replaced by highly specialized stem cells from a healthy donor. The best outcome is achieved when stem cells come from a genetically matched sibling. If the patient receives stem cells from someone else, immunity will be partially restored and the patient may continue to experience complex medical problems.
In the latest research, NIAID tested the safety and effectiveness of gene therapy and found that inserting a normal IL2RG gene alongside low-dose chemotherapy helped patients to rebuild immune system over time. The research involved five SCID-XI patients of age 7 to 24 who suffered worsening immune system despite one or more previous transplants from a parent.
In the experimental treatment, lentiviral gene transfer treatment was used. The patient’s bone was extracted and normal IL2RG gene was delivered. Then, with a lose dose of chemotherapy, corrected cells were infused back into the patient which developed into healthy bone marrow and begin producing new blood cells.
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According to the report, two patients who received gene therapy showed significant improvement in immunity with one patient continuing to improve after three years of therapy while the other one died of a pre-existing infection and lung damage two years after the therapy. The remaining three received therapy three to six months ago and they are also showing improvement in their immune systems. Surviving patients will continue to be monitored in the years to come.