Several researchers have come up with a new way to treat rare genetic disorder through advanced gene-splicing. New gene therapy rebuilds immune response, greatly improving the patient’s chances of survival.
For some time now, doctors have employed gene therapy in order to treat severe genetical abnormalities. Although most cases require more than your simple run-of-the-mill viral vectoring, doctors are confident that they will be able to develop advanced gene therapy in order to help out those who were born with genetic disorders.
The group of researchers were looking for a way to improve treatment for patients who suffer from a specific type of immune deficiency, known as SCID-X1. Normally, this type of affliction is very rare, affecting 1 in 100000. Furthermore, studies have revealed that male children are most likely to exhibit this type of genetical abnormality.
More on the illness. According to the medical literature on the matter, patients who are born with this genetic abnormality are incapable of producing white cells. Which means that their bodies are incapable of fending off pathogens, leaving the body susceptible to multiple infections. Furthermore, the researchers have retraced the problem to a defective gene, or rather a mutated variant of the IL2RG gene. People born with this defective gene are more susceptible to infections than people with a normally developed immune system.
Prognosis states that the new gene therapy rebuilds immune response. But more on that later. Naturally, this type of genetic disorder is countered using a transplant of fresh stem cells, harvested from a child’s parent. Research have shown that if the child has any siblings, his chances of survival and building up an immune response will increase. Relying on the stem cells harvested from his parent only gives him a 50/50 chance of actually displaying some improvement.
But the doctors are not easily swayed. The novel treatment employs the use of low dose chemotherapy along with gene therapy in orde to treat rare genetical abnormalities. Five volunteers were drafted for this medical trial. Their ages ranged between 7 and 24 years old. The doctors from NIAID harvested stem cells from the parents. After that, they genetically engineered a Lentivirus, in order to provide a good delivery vector. The virus was then tasked with identifying and delivering the right gene to the right cell. After the virus finished the job, the doctors administered low doses of chemotherapy in order to wash out the defective cells. Then, if the stem cells displayed the cored IL2RG gene, they were reintroduced into the blood stream.
After the transplant was performed, all members participating in this trial have shown signs of improvement.