![]() Five of six patients who were followed for at least 24 months experienced cellular immune reconstruction at a median of 12 months (range, 6-24).Īlso, there was an increase in levels of CD3+, CD4+ and CD8+ T cells, naive CD4+ T cells and regulatory T cells for a 9-month period following infusion. The researchers detected gene-marked CD3+ cells at a median of 12 weeks (range, 6-16) in all 10 infants. Median follow-up was 31.2 months (range, 10-48.9). Patients underwent monthly evaluations through month 6, then every 3 months through month 24. The researchers began measuring the patients’ immune reconstitution and gene marking 4 weeks after infusion and T-cell receptor (TCR) diversity and vector insertion-site analysis 12 weeks after infusion. The median age at infusion of gene-transduced cells was 2.7 months (range, 2.3-13.3). ![]() The patients then received IV busulfan including these cells over a period of 2 days. The study involved 10 infants (60% boys) who did not have an HLA-matched sibling enrolled and treated at UCSF Benioff Children’s Hospital for newly diagnosed ART-SCID between June 2018 and September 2021.Ĭlinicians manufactured, cryopreserved and performed quality-control testing of the patients’ gene-transduced autologous CD34+ cells, which in effect included corrected copies of the DCLRE1C gene. “Gene therapy, achieved by adding a correct copy of the Artemis gene to the patient’s own blood-forming stem cells harvested from their bone marrow, avoids the complications of rejection and graft-versus-host disease and offers patients with ART-SCID the chance for a long and healthy life,” Puck said. Cowan, MD, pediatric immunologist at UCSF Benioff Children’s Hospital, told Healio. This defect in their DNA also makes them more sensitive to chemotherapy and radiation therapy than other patients, Morton J. S ource: Adobe StockĪRT-SCID responds poorly to typical SCID treatment, allogenic hematopoietic stem cell transplantation (HSCT), even when it involves sibling donors with matching human leukocyte antigens (HLA), according to the researchers. The gene therapy adds a healthy copy of the DCLRE1C gene back into the bodies of infants with Artemis-deficiency severe combined immunodeficiency to stimulate the growth of T cells and B cells. Mutations in the DNA cross-link repair 1C ( DCLRE1C) gene lead to Artemis-deficient severe combined immunodeficiency (ART-SCID), which is characterized by a lack of T lymphocytes with a lack of or nonfunctional B lymphocytes. Infants with Artemis-deficient severe combined immunodeficiency improved after corrected genes in their stem cells enabled them to grow their own T cells and B cells, according to a study published in The New England Journal of Medicine. If you continue to have this issue please contact to Healio
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