"The high overall survival and event-free survival observed at 24 and 36 months compare favorably with survival that has been observed previously with HSCT, the current standard of care for the treatment of ADA-SCID," the authors write. studies discontinued immunoglobulin-replacement therapy by 24 and 36 months, respectively. Immune reconstitution was robust 90 and 100 percent of those in the U.S. Sustained metabolic detoxification and normalization of ADA activity levels were observed in patients. study, engraftment of genetically modified HSPCs persisted. study, respectively, at 12 months 97 and 95 percent, respectively, at 24 months and 95 percent in the U.K. Event-free survival was 97 and 100 percent in the U.S. The researchers found that in all studies, overall survival was 100 percent.
studies with 24 months of follow-up and 20 patients in a U.K. Data were included from 30 patients in two U.S. Kohn, M.D., from the University of California, Los Angeles, and colleagues treated 50 patients with ADA-SCID with investigational gene therapy. TUESDAY, (HealthDay News) - For patients with severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID), gene therapy composed of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a self-inactivating lentiviral vector encoding human ADA results in high overall and event-free survival, according to a study published online May 11 in the New England Journal of Medicine.ĭonald B. Editors and writers make all efforts to clarify any financial ties behind the studies on which we report. All of our articles are chosen independent of any financial interests. HeathDay is committed to maintaining the highest possible levels of impartial editorial standards in the content that we present on our website. Any known potential conflicts of interest associated with a study or source are made clear to the reader.Įditorial and Fact-Checking Policy for more detail.Įditorial and Fact-Checking Policy HealthDay Editorial Commitment.Each article includes a link or reference to the original source.Peer-reviewed journals or issued from independent and respected medical associations, academic groups and governmental organizations. Unless otherwise noted, all articles focusing on new research are based on studies published in.All articles are edited and checked for factual accuracy by our.Team of industry experts to ensure accuracy. Our syndicated news content is completely independent of any financial interests, is based solely on industry-respected sources and the latest scientific research, and is carefully fact-checked by a HealthDay operates under the strictest editorial standards.
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