Broad-spectrum antibodies against self-antigens and cytokines in RAG deficiency.

نویسندگان

  • Jolan E Walter
  • Lindsey B Rosen
  • Krisztian Csomos
  • Jacob M Rosenberg
  • Divij Mathew
  • Marton Keszei
  • Boglarka Ujhazi
  • Karin Chen
  • Yu Nee Lee
  • Irit Tirosh
  • Kerry Dobbs
  • Waleed Al-Herz
  • Morton J Cowan
  • Jennifer Puck
  • Jack J Bleesing
  • Michael S Grimley
  • Harry Malech
  • Suk See De Ravin
  • Andrew R Gennery
  • Roshini S Abraham
  • Avni Y Joshi
  • Thomas G Boyce
  • Manish J Butte
  • Kari C Nadeau
  • Imelda Balboni
  • Kathleen E Sullivan
  • Javeed Akhter
  • Mehdi Adeli
  • Reem A El-Feky
  • Dalia H El-Ghoneimy
  • Ghassan Dbaibo
  • Rima Wakim
  • Chiara Azzari
  • Paolo Palma
  • Caterina Cancrini
  • Kelly Capuder
  • Antonio Condino-Neto
  • Beatriz T Costa-Carvalho
  • Joao Bosco Oliveira
  • Chaim Roifman
  • David Buchbinder
  • Attila Kumanovics
  • Jose Luis Franco
  • Tim Niehues
  • Catharina Schuetz
  • Taco Kuijpers
  • Christina Yee
  • Janet Chou
  • Michel J Masaad
  • Raif Geha
  • Gulbu Uzel
  • Rebecca Gelman
  • Steven M Holland
  • Mike Recher
  • Paul J Utz
  • Sarah K Browne
  • Luigi D Notarangelo
چکیده

Patients with mutations of the recombination-activating genes (RAG) present with diverse clinical phenotypes, including severe combined immune deficiency (SCID), autoimmunity, and inflammation. However, the incidence and extent of immune dysregulation in RAG-dependent immunodeficiency have not been studied in detail. Here, we have demonstrated that patients with hypomorphic RAG mutations, especially those with delayed-onset combined immune deficiency and granulomatous/autoimmune manifestations (CID-G/AI), produce a broad spectrum of autoantibodies. Neutralizing anti-IFN-α or anti-IFN-ω antibodies were present at detectable levels in patients with CID-G/AI who had a history of severe viral infections. As this autoantibody profile is not observed in a wide range of other primary immunodeficiencies, we hypothesized that recurrent or chronic viral infections may precipitate or aggravate immune dysregulation in RAG-deficient hosts. We repeatedly challenged Rag1S723C/S723C mice, which serve as a model of leaky SCID, with agonists of the virus-recognizing receptors TLR3/MDA5, TLR7/-8, and TLR9 and found that this treatment elicits autoantibody production. Altogether, our data demonstrate that immune dysregulation is an integral aspect of RAG-associated immunodeficiency and indicate that environmental triggers may modulate the phenotypic expression of autoimmune manifestations.

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عنوان ژورنال:
  • The Journal of clinical investigation

دوره 125 11  شماره 

صفحات  -

تاریخ انتشار 2015