Article
Ruxolitinib reverses dysregulated T helper cell responses and controls autoimmunity caused by a novel signal transducer and activator of transcription 1 (STAT1) gain-of-function mutation.
The Journal of allergy and clinical immunology - 1 May 2017
Weinacht Katja G, Charbonnier Louis-Marie, Alroqi Fayhan, Plant Ashley, Qiao Qi, Wu Hao, Ma Clement, Torgerson Troy R, Rosenzweig Sergio D, Fleisher Thomas A, Notarangelo Luigi D, Hanson Imelda C, Forbes Lisa R, Chatila Talal A
Abstract excerpt
BACKGROUND: Gain-of-function (GOF) mutations in the human signal transducer and activator of transcription 1 (STAT1) manifest in immunodeficiency and autoimmunity with impaired TH17 cell differentiation and exaggerated responsiveness to type I and II interferons. Allogeneic bone marrow transplantation has been attempted in severely affected patients, but outcomes have been poor. OBJECTIVE: We sought to define the...
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