[HTML][HTML] Partial V (D) J recombination activity leads to Omenn syndrome

A Villa, S Santagata, F Bozzi, S Giliani, A Frattini… - Cell, 1998 - cell.com
A Villa, S Santagata, F Bozzi, S Giliani, A Frattini, L Imberti, LB Gatta, HD Ochs, K Schwarz…
Cell, 1998cell.com
Genomic rearrangement of the antigen receptor loci is initiated by the two lymphoid-specific
proteins Rag-1 and Rag-2. Null mutations in either of the two proteins abrogate initiation of V
(D) J recombination and cause severe combined immunodeficiency with complete absence
of mature B and T lymphocytes. We report here that patients with Omenn syndrome, a
severe immunodeficiency characterized by the presence of activated, anergic, oligoclonal T
cells, hypereosinophilia, and high IgE levels, bear missense mutations in either the Rag-1 or …
Abstract
Genomic rearrangement of the antigen receptor loci is initiated by the two lymphoid-specific proteins Rag-1 and Rag-2. Null mutations in either of the two proteins abrogate initiation of V(D)J recombination and cause severe combined immunodeficiency with complete absence of mature B and T lymphocytes. We report here that patients with Omenn syndrome, a severe immunodeficiency characterized by the presence of activated, anergic, oligoclonal T cells, hypereosinophilia, and high IgE levels, bear missense mutations in either the Rag-1 or Rag-2 genes that result in partial activity of the two proteins. Two of the amino acid substitutions map within the Rag-1 homeodomain and decrease DNA binding activity, while three others lower the efficiency of Rag-1/Rag-2 interaction. These findings provide evidence to indicate that the immunodeficiency manifested in patients with Omenn syndrome arises from mutations that decrease the efficiency of V(D)J recombination.
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