![]() ![]() 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. 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. study) at 12 months 97% and 95%, respectively, at 24 months and 95% (U.K. Event-free survival (in the absence of reinitiation of enzyme-replacement therapy or rescue allogeneic hematopoietic stem-cell transplantation) was 97% (U.S. Overall survival was 100% in all studies up to 24 and 36 months. study (in which a fresh formulation was used) at 36 months of follow-up. studies (in which fresh and cryopreserved formulations were used) at 24 months of follow-up were analyzed alongside data from the U.K. We treated 50 patients with ADA-SCID (30 in the United States and 20 in the United Kingdom) with an investigational gene therapy composed of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a self-inactivating lentiviral vector encoding human ADA. Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency. Original Article Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency List of authors. The most trusted, influential source of new medical knowledge and clinical best practices in the world. Information and tools for librarians about site license offerings. Valuable tools for building a rewarding career in health care. The authorized source of trusted medical research and education for the Chinese-language medical community. The most advanced way to teach, practice, and assess clinical reasoning skills. ![]() Information, resources, and support needed to approach rotations - and life as a resident. The most effective and engaging way for clinicians to learn, improve their practice, and prepare for board exams. NEW! Peer-reviewed journal featuring in-depth articles to accelerate the transformation of health care delivery.Ĭoncise summaries and expert physician commentary that busy clinicians need to enhance patient care. NEW! A digital journal for innovative original research and fresh, bold ideas in clinical trial design and clinical decision-making. ![]()
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