Blood published online (12 March 2010).Ĭartier, N. Gatti, R.A., Meuwissen, H.J., Allen, H.D., Hong, R. Again, SCID seemed to be the best condition in which to test the feasibility of this new approach, which led to the first clinical trial 20 years ago.īuckley, R.H. This situation is what prompted the emergence of the concept of gene therapy 3, together with the known properties of retroviruses 4. In the absence of an HLA-identical donor, transplantation of allogeneic HSCs, however, is still marred by the devastating consequences of graft-versus-host disease. Remarkably, the success of transplantation of HLA-identical bone marrow into patients with SCID did not require the destruction of host stem cells. 2), which paved the way to the development of transplantation of allogeneic hematopoietic stem cells (HSCs) for a much wider range of conditions. Given the absence of T cells in these patients, it was thought that SCID sufferers could not reject a graft. In addition, one form of SCID became the first human illness treated by human gene therapy in 1990, a process in which a normal gene was transferred into the defective white blood cells of two young girls to compensate for the genetic mutation. Once the human leukocyte antigen (HLA) system and its involvement in allogeneic responses were progressively deciphered in the 1950s and 1960s, it became conceivable to cure babies with SCID by transplantation of HLA-identical bone marrow cells. Meanwhile, the term 'SCID' became very popular in the immunological community, as natural SCID mouse mutants were discovered and were widely used for the study of lymphocyte ontogeny and to serve as recipients for allogeneic and, above all, xenogeneic (human) grafts. The molecular analysis of these conditions generated a flood of information on key steps in lymphocyte development 1. It was subsequently recognized that SCID covers a variety of genetic defects, all of which lead to profoundly impaired differentiation of T lymphocytes and (in some cases) additional blocks in the differentiation of B lymphocytes and/or natural killer (NK) lymphocytes. More than 50 years ago, the term 'severe combined immunodeficiency' (SCID) was coined to designate rare, lethal conditions in which infants die from an array of infections associated with a lack of lymphocytes in the blood.
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