JAK inhibition in a patient with a STAT1 gain-of-function variant reveals STAT1 dysregulation as a common feature of aplastic anemia

Idiopathic aplastic anemia is a potentially lethal disease, characterized by T cell-mediated autoimmune attack of bone marrow hematopoietic stem cells. Standard of care therapies (stem cell transplantation or immunosuppression) are effective but associated with a risk of serious toxicities. An 18-ye...

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Veröffentlicht in:	Med (New York, N.Y. : Online) N.Y. : Online), 2022-01, Vol.3 (1), p.42-57.e5
Hauptverfasser:	Rosenberg, Jacob M., Peters, Joshua M., Hughes, Travis, Lareau, Caleb A., Ludwig, Leif S., Massoth, Lucas R., Austin-Tse, Christina, Rehm, Heidi L., Bryson, Bryan, Chen, Yi-Bin, Regev, Aviv, Shalek, Alex K., Fortune, Sarah M., Sykes, David B.
Format:	Artikel
Sprache:	eng
Schlagworte:	Acetonitriles Adolescent Anemia, Aplastic - genetics Aplastic anemia autoimmune CD8-Positive T-Lymphocytes Gain of Function Mutation Humans interferon gamma itacitinib JAK inhibition JAK/STAT Janus Kinase Inhibitors - pharmacology Male Pyrazoles Pyrimidines Pyrroles STAT1 gain-of-function STAT1 GOF STAT1 Transcription Factor - genetics T-cell exhaustion
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