Article
Recapitulating primary immunodeficiencies with expanded potential stem cells: Proof of concept with STAT1 gain of function.
The Journal of allergy and clinical immunology - 1 Apr 2024
Liu Xueyan, Chan Vera S F, Smith Kenneth G C, Ming Chang, Or Chung Sze, Tsui Faria T W, Gao Bo, Cook Matthew C, Liu Pentao, Lau Chak Sing, Li Philip Hei
Abstract excerpt
BACKGROUND: Inborn errors of immunity (IEI) often lack specific disease models and personalized management. Signal transducer and activator of transcription (STAT)-1 gain of function (GoF) is such example of an IEI with diverse clinical phenotype with unclear pathomechanisms and unpredictable response to therapy. Limitations in obtaining fresh samples for functional testing and research further highlights the...
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