Article
[Gene therapy of SCID-X1].
Bundesgesundheitsblatt, Gesundheitsforschung, Gesundheitsschutz - 1 Dec 2007
Baum C, Schambach A, Modlich U, Thrasher A
Abstract excerpt
X-linked severe combined immunodeficiency (SCID-X1) is an inherited disease caused by inactivating mutations in the gene encoding the interleukin 2 receptor common gamma chain (IL2RG), which is located on the X-chromosome. Affected boys fail to develop two major effector cell types of the immune system (T cells and NK cells) and suffer from a functional B cell defect. Although drugs such as antibiotics can offer...
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