Researchers found that after testing five SCID-X1 patients who received both gene therapy and low-dose chemotherapy, four of the patients showed improvements in immune function. Scientists from the National Institute of Allergy and Infectious Diseases (NIAID) have come up with a new gene therapy that can restore immunity…
The standard treatment would include a transplant of stem cells, which can be obtained from the direct family members. Only one of them unfortunately perished, due to a pre-existing condition that damaged their lungs before the new therapy could be applied. Currently, patients suffering from SCID-X1 are treated with stem cell transplants.
The National Institute of Allergy and Infectious Diseases reports a procedure including the extraction, genetic correction and reinsertion of a patient’s bone marrow can help their immune systems improve over time.
SCID-X1 is generally triggered by mutations in the IL2RG gene that averts infection-fighting immune cells from developing and working normally, leaving inflicted children highly vulnerable to life-threatening infections. One of the lifesaving treatments for very young children with SCID-X1 is transplantation of stem cells, ideally from a sibling donor who is genetically matched.
In the latest study, scientists examined the safety and effectiveness of gene therapy pooled with low-dose chemotherapy in five SCID-X1 patients.
Using a lentiviral vector, the scientists included a healthy IL2RG gene in the stem cells. The patients were then given low-dose chemotherapy to eliminate the defective blood cells. One patient received treatment three years ago with continued improvements. Another patient is doing well and continues to show improvement even after three years of the gene therapy. Three more patients aged 7 to 24 are improving, though it has only been three to six months since the treatment was performed. All surviving patients are continually being monitored.
Severe Combined Immunodeficiency is a rare immunodeficiency that leads to frequent serious infections. They manipulated the virus gene to deliver the right gene to the cell. By collecting an individual s stem cells and modifying them with a lentivirus, the gene-corrected cells can be returned into the blood to help produce normal healthy immune cells. Accessible treatments for this once-deadly disorder have centered around bone marrow transplants, yet encouraging results have been reported by an on-going clinical trial for an emerging gene therapy – Lentiviral gene transfer.
To determine the safety and effectiveness of lentiviral gene transfer as a treatment for children and adolescents with X-linked severe combined immunodeficiency.
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