Mendelian susceptibility to mycobacterial disease: genetic, immunological, and clinical features of inborn errors of IFN-γ immunity

J Bustamante, S Boisson-Dupuis, L Abel… - Seminars in …, 2014 - Elsevier
Mendelian susceptibility to mycobacterial disease (MSMD) is a rare condition characterized
by predisposition to clinical disease caused by weakly virulent mycobacteria, such as BCG …

Human STAT1 Gain-of-Function Heterozygous Mutations: Chronic Mucocutaneous Candidiasis and Type I Interferonopathy

S Okada, T Asano, K Moriya, S Boisson-Dupuis… - Journal of clinical …, 2020 - Springer
Heterozygous gain-of-function (GOF) mutations in STAT1 in patients with chronic
mucocutaneous candidiasis (CMC) and hypothyroidism were discovered in 2011. CMC is …

Heterozygous STAT1 gain-of-function mutations underlie an unexpectedly broad clinical phenotype

J Toubiana, S Okada, J Hiller… - Blood, The Journal …, 2016 - ashpublications.org
Since their discovery in patients with autosomal dominant (AD) chronic mucocutaneous
candidiasis (CMC) in 2011, heterozygous STAT1 gain-of-function (GOF) mutations have …

JAKs and STATs from a clinical perspective: loss-of-function mutations, gain-of-function mutations, and their multidimensional consequences

N Ott, L Faletti, M Heeg, V Andreani… - Journal of clinical …, 2023 - Springer
The JAK/STAT signaling pathway plays a key role in cytokine signaling and is involved in
development, immunity, and tumorigenesis for nearly any cell. At first glance, the JAK/STAT …

Chronic mucocutaneous candidiasis disease associated with inborn errors of IL‐17 immunity

S Okada, A Puel, JL Casanova… - Clinical & translational …, 2016 - Wiley Online Library
Chronic mucocutaneous candidiasis (CMC) is characterized by recurrent or persistent
infections affecting the nails, skin and oral and genital mucosae caused by Candida spp …

Ruxolitinib reverses dysregulated T helper cell responses and controls autoimmunity caused by a novel signal transducer and activator of transcription 1 (STAT1) gain …

KG Weinacht, LM Charbonnier, F Alroqi, A Plant… - Journal of Allergy and …, 2017 - Elsevier
Background Gain-of-function (GOF) mutations in the human signal transducer and activator
of transcription 1 (STAT1) manifest in immunodeficiency and autoimmunity with impaired TH …

Inherited IL-17RC deficiency in patients with chronic mucocutaneous candidiasis

Y Ling, S Cypowyj, C Aytekin, M Galicchio… - Journal of Experimental …, 2015 - rupress.org
Chronic mucocutaneous candidiasis (CMC) is characterized by recurrent or persistent
infections of the skin, nail, oral, and genital mucosae with Candida species, mainly C …

Genetic, immunological, and clinical features of patients with bacterial and fungal infections due to inherited IL-17RA deficiency

R Lévy, S Okada, V Béziat, K Moriya… - Proceedings of the …, 2016 - National Acad Sciences
Chronic mucocutaneous candidiasis (CMC) is defined as recurrent or persistent infection of
the skin, nails, and/or mucosae with commensal Candida species. The first genetic etiology …

Hematopoietic stem cell transplantation in patients with gain-of-function signal transducer and activator of transcription 1 mutations

JW Leiding, S Okada, D Hagin, M Abinun… - Journal of Allergy and …, 2018 - Elsevier
Background Gain-of-function (GOF) mutations in signal transducer and activator of
transcription 1 (STAT1) cause susceptibility to a range of infections, autoimmunity, immune …

Flow cytometry-based diagnosis of primary immunodeficiency diseases

H Kanegane, A Hoshino, T Okano, T Yasumi… - Allergology …, 2018 - jstage.jst.go.jp
abstract Primary immunodeficiencies (PIDs) are a heterogeneous group of inherited
diseases of the immune system. The definite diagnosis of PID is ascertained by genetic …