- Walter, Jolan E;
- Rucci, Francesca;
- Patrizi, Laura;
- Recher, Mike;
- Regenass, Stephan;
- Paganini, Tiziana;
- Keszei, Marton;
- Pessach, Itai;
- Lang, Philipp A;
- Poliani, Pietro Luigi;
- Giliani, Silvia;
- Al-Herz, Waleed;
- Cowan, Morton J;
- Puck, Jennifer M;
- Bleesing, Jack;
- Niehues, Tim;
- Schuetz, Catharina;
- Malech, Harry;
- DeRavin, Suk See;
- Facchetti, Fabio;
- Gennery, Andrew R;
- Andersson, Emma;
- Kamani, Naynesh R;
- Sekiguchi, JoAnn;
- Alenezi, Hamid M;
- Chinen, Javier;
- Dbaibo, Ghassan;
- ElGhazali, Gehad;
- Fontana, Adriano;
- Pasic, Srdjan;
- Detre, Cynthia;
- Terhorst, Cox;
- Alt, Frederick W;
- Notarangelo, Luigi D
The contribution of B cells to the pathology of Omenn syndrome and leaky severe combined immunodeficiency (SCID) has not been previously investigated. We have studied a mut/mut mouse model of leaky SCID with a homozygous Rag1 S723C mutation that impairs, but does not abrogate, V(D)J recombination activity. In spite of a severe block at the pro-B cell stage and profound B cell lymphopenia, significant serum levels of immunoglobulin (Ig) G, IgM, IgA, and IgE and a high proportion of Ig-secreting cells were detected in mut/mut mice. Antibody responses to trinitrophenyl (TNP)-Ficoll and production of high-affinity antibodies to TNP-keyhole limpet hemocyanin were severely impaired, even after adoptive transfer of wild-type CD4(+) T cells. Mut/mut mice produced high amounts of low-affinity self-reactive antibodies and showed significant lymphocytic infiltrates in peripheral tissues. Autoantibody production was associated with impaired receptor editing and increased serum B cell-activating factor (BAFF) concentrations. Autoantibodies and elevated BAFF levels were also identified in patients with Omenn syndrome and leaky SCID as a result of hypomorphic RAG mutations. These data indicate that the stochastic generation of an autoreactive B cell repertoire, which is associated with defects in central and peripheral checkpoints of B cell tolerance, is an important, previously unrecognized, aspect of immunodeficiencies associated with hypomorphic RAG mutations.