Ten children with a rare condition had their immune systems restored with gene-replacement therapy.
The person is Grace Wade.
There is a rare genetic disorder called Artemis- deficient SCID.
The Polaris/eyevine Copyright is owned by Santiago Mejia.
A new gene-replacement therapy may be able to lead normal lives for children with a rare genetic disorder. The therapy restored the immune systems of 10 babies with the condition.
Between 40 and 100 babies in the US are diagnosed with the disease. Bubble boy disease is a condition in which a child with a lack of a functioning immune system is forced to live in a plastic bubble. Unless a bone marrow transplant is performed, most children with SCID will die before they are 2 years old. Babies with a rare form of the condition are less likely to have a successful transplant.
The University of California, San Francisco and their colleagues wanted to see if gene-replacement therapy could be used to treat Artemis- deficient SCID.
The defect in the gene that codes for Artemis causes this type of SCID. T cells and B cells can't be produced without Artemis. Stem cells were taken from the bone marrow of 10 infants with the condition. Children were given a low dose of chemotherapy to kill their bone marrow cells. The corrected stem cells were infused into them with an IV.
All the children produced T cells and B cells after treatment. Five infants who received the therapy two or more years ago now have functioning immune systems. The other participants should also develop fully functional immune systems with time. There will be trials with more children.
There were no serious side effects from the treatment, but researchers plan to follow the children for a long time. The study's small sample size may have made it miss other possible side effects. The treatment is an advancement in treating Artemis- deficient SCID.
The New England Journal of Medicine has a journal title.
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