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Jakinibs for the Treatment of Immunodysregulation in Patients with Gain of Function STAT1 or STAT3 Mutations
Lisa R. Forbes ,
Tiphanie P. Vogel ,
Megan A. Cooper ,
Johana Castro-Wagner ,
Edith Schussler ,
Katja G. Weinacht ,
Ashley S. Plant ,
Helen C. Su ,
Eric J. Allenspach ,
Mary Slatter ,
Mario Abinun ,
Desa Lilic ,
Charlotte Cunningham-Rundles ,
Olive Eckstein ,
Peter Olbrich ,
R. Paul Guillerman ,
Niraj C. Patel ,
Yesim Y. Demirdag ,
Christa Zerbe ,
Alexandra F. Freeman ,
Steven M. Holland ,
Paul Szabolcs ,
Andrew Gennery ,
Troy R. Torgerson ,
Joshua D. Milner ,
Jennifer W. Leiding
August 2018
August 2018
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Abstract
<p id="P2">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
<i>STAT1</i> GOF or
<i>STAT3</i> GOF mutations.
</p>
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