Marie-Louise Frémond
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Etude des voies de signalisation de l’interféron de type I de cellules du système nerveux central déficientes en TREX1 dérivées de cellules souches pluripotentes inductibles humaines.
Open Date: 2015-11-01
Close Date: 2018-10-01
Articles (3)
Juvenile idiopathic arthritis (JIA) is a heterogeneous group of diseases with probably differential underlying physiopathology. Despite the revolutionary era of biologics, some patients remain difficult to treat because of disease severity, drug adverse events, drug allergy or association with severe comorbidities, i.e., uveitis, interstitial lung disease and macrophagic activation syndrome. Janus Kinase (JAK) inhibitors are small molecules that target JAK/Signal Transducers and Activators of Transcription (STAT) pathways, which could then prevent the activity of several proinflammatory cytokines. They may provide a useful alternative in these cases of JIA or in patients actually affected by Mendelian disorders mimicking JIA, such as type I interferonopathies with joint involvement, and might be the bridge for haematopoietic stem cell transplantation in these disabling conditions. As these treatments may have side effects that should not be ignored, ongoing and further controlled studies are still needed to provide data underlying long-term safety considerations in children and delineate subsets of JIA patients that will benefit from these promising treatments.
Year:
2023
Year:
2020
Year:
2020
Collaborators (2)
Isabelle Melki
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Caroline Schmitt
Associate professor
INSERM U1148
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