The corrected cells are then returned to the child’s body, where they are intended to produce a continual supply of healthy immune cells capable of fighting infection. Next, by means of an approach developed by the research team, a new copy of the ADA gene is delivered into the stem cells by a modified lentivirus, or viral vector. The gene therapy that was evaluated in the study begins with the collection of some of the child’s blood-forming stem cells, which have the potential to create all types of blood and immune cells. No evidence of monoclonal expansion, leukoproliferative complications, or emergence of replication-competent lentivirus was noted, and no events of autoimmunity or graft-versus-host disease occurred. study discontinuing immunoglobulin-replacement therapy by 24 months and 36 months, respectively. “Immune reconstitution was robust, with 90% of the patients in the U.S. “Patients had sustained metabolic detoxification and normalization of ADA activity levels.
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studies and in 19 of 20 patients in the U.K. “Engraftment of genetically modified HSPCs persisted in 29 of 30 patients in the U.S. The article’s authors, led by UCLA’s Donald Kohn, MD, and GOSH’s Claire Booth, MB, BS, PhD, detailed the two- and three-year outcomes for children treated with the investigational lentiviral gene therapy in clinical trials at GOSH, UCLA Mattel Children’s Hospital, and the National Institutes of Health between 20. According to an article (“ Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency”) that appeared May 11 in the New England Journal of Medicine, 48 of the 50 children retain replenished immune system function two to three years after receiving treatment. Besides curing dreadful diseases, gene therapy can help patients avoid the complications of alternative treatments such as transplants of donor-provided stem cells (which carry risks such as graft-versus-host disease and side effects from chemotherapy) and enzyme replacement therapy (which does not fully reconstitute immune function and must be taken for life, usually once or twice weekly).Ĭora Oakley numbers among 50 children who were recently evaluated for their response to an experimental gene therapy that was developed by a team of researchers from UCLA and Great Ormond Street Hospital (GOSH) in London. The Oakley’s experience dramatizes how autologous gene therapy can be doubly advantageous. It still shocks me all the time how unbelievably fortunate we are.” And then here’s my daughter who had this life-changing treatment that felt like a miracle.
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“I just thought no one should have to suffer like this. “I saw young bone marrow transplant patients who developed graft-versus-host disease and others who had to take all these antirejection medications and still had issues,” recalled Chelsea Oakley.