Article
Complete correction of murine Artemis immunodeficiency by lentiviral vector-mediated gene transfer
24 Oct 2006
Abstract excerpt
Artemis gene mutations are responsible for the development of a severe combined immunodeficiency [radiation-sensitive (RS) SCID] characterized by a severe B and T cell deficiency and a normal natural killer cell population. To establish the feasibility of a gene therapy approach to the treatment of RS-SCID, we generated a series of lentiviral vectors expressing human Artemis from different promoters and used them...
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