![]() ![]() T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. ![]() ![]() Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID). Radiosensitive severe combined immunodeficiency disease. Long-term health-related outcomes in survivors of childhood cancer treated with HSCT versus conventional therapy: a report from the Bone Marrow Transplant Survivor Study (BMTSS) and Childhood Cancer Survivor Study (CCSS). Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? J Allergy Clin Immunol. How I treat severe combined immunodeficiency. Severe combined immunodeficiency (SCID) in Canadian children: a national surveillance study. A founder mutation in Artemis, an SNM1-like protein, causes SCID in Athabascan-speaking Native Americans. Current progress on gene therapy for primary immunodeficiencies. Papers of particular interest, published recently, have been highlighted as: Looking to the future, gene editing strategies hold enormous promise, and SCID will continue to be the paradigm condition for the development of these applications. With safer approaches, we anticipate other SCIDs such as recombinase activating gene and Artemis deficiency will be treated with HSC-GT. Clinical trials using self-inactivating γ-retroviral and lentiviral vectors incorporating improved safety features are currently being conducted in Europe and North America. Initial clinical trials employed γ-retroviral vectors, but leukaemia arose in five SCID-X1 patients due to insertional mutagenesis. SCID-X1 and adenosine deaminase SCID have been successfully treated with hematopoietic stem cell gene therapy (HSC-GT) and patients show impressive levels of immune reconstitution. They have been identified as ideal candidates for treatment by gene therapy and have led the development of gene therapy for other bone marrow disorders. Severe combined immunodeficiencies (SCIDs) are a group of rare genetic diseases characterized by profound abnormalities of cellular and humoral immunity. ![]()
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