TY - JOUR AU - Forbes, Lisa R AU - Vogel, Tiphanie P AU - Cooper, Megan A AU - Castro-Wagner, Johana AU - Schussler, Edith AU - Weinacht, Katja G AU - Plant, Ashley S AU - Su, Helen C AU - Allenspach, Eric J AU - Slatter, Mary AU - Abinun, Mario AU - Lilic, Desa AU - Cunningham-Rundles, Charlotte AU - Eckstein, Olive AU - Olbrich, Peter AU - Guillerman, R Paul AU - Patel, Niraj C AU - Demirdag, Yesim Y AU - Zerbe, Christa AU - Freeman, Alexandra F AU - Holland, Steven M AU - Szabolcs, Paul AU - Gennery, Andrew AU - Torgerson, Troy R AU - Milner, Joshua D AU - Leiding, Jennifer W PY - 2018 DO - 10.1016/j.jaci.2018.07.020 UR - http://hdl.handle.net/10668/12815 T2 - The Journal of allergy and clinical immunology AB - Treatment of the autoimmune and immune-dysregulatory features of patients with STAT1 GOF or STAT3 GOF disease remains challenging. Jakinibs have been used to treat the severe immune-dysregulation in patients with either STAT1 GOF or STAT3 GOF mutations. LA - en KW - Adolescent KW - Adult KW - Child KW - Female KW - Gain of Function Mutation KW - Humans KW - Immunologic Deficiency Syndromes KW - Janus Kinases KW - Male KW - Nitriles KW - Piperidines KW - Protein Kinase Inhibitors KW - Pyrazoles KW - Pyrimidines KW - Pyrroles KW - STAT1 Transcription Factor KW - STAT3 Transcription Factor KW - Treatment Outcome TI - Jakinibs for the treatment of immune dysregulation in patients with gain-of-function signal transducer and activator of transcription 1 (STAT1) or STAT3 mutations. TY - research article VL - 142 ER -