Pharmacological evaluation of drug therapies in Aicardi-Goutières syndrome: insights from patient-derived neural stem cells

Pharmacological evaluation of drug therapies in Aicardi-Goutières syndrome: insights from patient-derived neural stem cells

Aicardi-Goutières syndrome (AGS) is a rare genetic disorder classified among type I interferonopathies. Current pharmacological management of AGS is symptomatic and supportive, with recent clinical applications of JAK inhibitors (JAKi) and antiretroviral therapies (RTIs). To investigate the effects...

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Main Authors: Stefania Braidotti, Rosalba Monica Ferraro, Raffaella Franca, Elena Genova, Francesco Giambuzzi, Andrea Mancini, Valentina Marinozzi, Letizia Pugnetti, Giulia Zudeh, Alessandra Tesser, Alberto Tommasini, Giuliana Decorti, Silvia Clara Giliani, Gabriele Stocco
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-03-01
Series:Frontiers in Pharmacology
Subjects:
patient-derived stem cell
Aicardi-Goutières syndrome
drug sensitivity
JAK inhibitors
antiretrovirals
Online Access:https://www.frontiersin.org/articles/10.3389/fphar.2025.1549183/full
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