Corrective gene transfer into bone marrow CD34+ cells for adenosine deaminase (ADA) deficiency: results in four patients after one year of follow-up. Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Persistence and expression of the adenosine deaminase gene for twelve years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial. American Society of Gene Therapy's Sixth Annual Meeting Executive Summaries, Vol. Immune Recovery Following Retroviral Mediated Common Gamma Chain Gene Therapy for X-linked Severe Combined Immunodeficiency. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 ((gamma) c deficiency). Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. Optimization of retroviral gene transfer protocol to maintain the lymphoid potential of progenitor cells. A defined window for efficient gene marking of severe combined immunodeficient-repopulating cells using a gibbon ape leukaemia virus-pseudotyped retroviral vector. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Nat Rev Immunol 2001 3: 200–208.Ĭavazzana-Calvo M et al. Primary immunodeficiency diseases: an experimental model for molecular medicine.
0 Comments
Leave a Reply. |
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |