Publication: Jakinibs for the treatment of immune dysregulation in patients with gain-of-function signal transducer and activator of transcription 1 (STAT1) or STAT3 mutations.
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Date
2018-08-06
Authors
Forbes, Lisa R
Vogel, Tiphanie P
Cooper, Megan A
Castro-Wagner, Johana
Schussler, Edith
Weinacht, Katja G
Plant, Ashley S
Su, Helen C
Allenspach, Eric J
Slatter, Mary
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Abstract
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.
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MeSH Terms
Adolescent
Adult
Child
Female
Gain of Function Mutation
Humans
Immunologic Deficiency Syndromes
Janus Kinases
Male
Nitriles
Piperidines
Protein Kinase Inhibitors
Pyrazoles
Pyrimidines
Pyrroles
STAT1 Transcription Factor
STAT3 Transcription Factor
Treatment Outcome
Adult
Child
Female
Gain of Function Mutation
Humans
Immunologic Deficiency Syndromes
Janus Kinases
Male
Nitriles
Piperidines
Protein Kinase Inhibitors
Pyrazoles
Pyrimidines
Pyrroles
STAT1 Transcription Factor
STAT3 Transcription Factor
Treatment Outcome