Health
SCID: Gene therapy restores immune system in child with rare disease
Ten children with a rare condition of severe combined immunodeficiency with Artemis deficiency had their immune systems partially or completely restored by gene replacement therapy
health
December 21, 2022
Artemis-deficient SCID is a rare genetic disorder Santiago Mejia/Polaris/eyevine Copyright: Santiago Mejia/Polaris/eyevine
Children born without a functioning immune system, due to a rare genetic disorder called Artemis-deficient severe combined immunodeficiency (Artemis-deficient SCID), develop new gene replacement therapyA trial found that treatment partially or completely restored the immune system of 10 infants with the condition.
Between 40 and 100 babies are diagnosed with SCID each year in the United States. Also known as Bubble Boy disease, it’s named after a 1970s documentary about a child who had to live inside a sterile plastic bubble because his immune system wasn’t working. Most children die before the age of two unless treated with a bone marrow transplant. However, infants with Artemis-deficient SCID (a rare subtype of the condition) are less likely to be successfully transplanted due to an inherent genetic defect.
Gene replacement therapy Treatment of other types of SCID, Morton Cowan UC San Francisco and his colleagues wanted to see if Artemis-deficient SCID could also be treated.
This subtype of SCID is caused by a defect in the gene encoding the protein Artemis.Without Artemis, the body cannot produce vital immune cells called T cells and B cells. stem cell They inserted the modified genetic information into cells from the bone marrow of 10 infants. The children then received low-dose chemotherapy to kill the cells in their bone marrow. This made room for the modified stem cells, which were injected into them using an IV.
Follow-up blood tests showed that all children produced T and B cells between 6 and 16 weeks after treatment. Of her six infants who were treated over two years ago, five now have fully functioning immune systems.Over time, other participants should also develop fully functioning immune systems, he says. jennifer pack, and the University of California, San Francisco. The next phase of research is to conduct trials on more children.
The treatment itself had no significant side effects, but researchers plan to follow the children longer to be sure, says Cowan. It is also possible that adverse side effects may have been overlooked. Vincent Bonagra at the Feinstein Institute of Medicine in New York. But ultimately, he says, the therapy is an important advance in the treatment of Artemis-deficient SCID.
Journal reference: New England Journal of Medicine, DOI: 10.1056/NEJMoa2206575
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