Severe autoinflammation in 4 patients with C-terminal variants in cell division control protein 42 homolog (CDC42) successfully treated with IL-1β inhibition

Affiliations

• ¹ Division of Allergy and Immunology, Department of Pediatrics, Stanford School of Medicine, Stanford, Calif. Electronic address: yaelg@stanford.edu.
• ² Translational Autoinflammatory Diseases Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, Md.
• ³ Department of Pediatric Rheumatology, University of Toronto and the Hospital for Sick Children, Toronto, Ontario, Canada.
• ⁴ Division of Human Gene Therapy, Department of Pediatrics, Stanford School of Medicine, Stanford, Calif.
• ⁵ Bioinformatics and Computational Biosciences Branch (BCBB) OCICB Rocky Mountain Laboratories, NIAID, NIH, Hamilton, Mont.
• ⁶ Division of Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
• ⁷ Broad Institute of MIT and Harvard, Cambridge, Mass.
• ⁸ Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
• ⁹ Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine, Stanford, Calif.
• ¹⁰ Division of Allergy and Immunology, Department of Pediatrics, Stanford School of Medicine, Stanford, Calif.
• ¹¹ Division of Rheumatology/RK Mellon Institute, Department of Pediatrics, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pa.
• ¹² Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine, Stanford, Calif. Electronic address: kgw1@stanford.edu.