Centre for Translational Stem Cell Biology, Hong Kong
Abstract: Inborn Errors of Immunity (IEI), with increased susceptibility to infections, autoimmune diseases, and malignancies but challenging to model and manage, is starving for deeper molecular underpinnings and novel therapeutic approaches. Signal transducer and activator of transcription-1 (STAT-1) gain of function (GoF) was previously identified as a crucial pathogenic feature in IEI. Based on our patient-derived expanded potential stem cells (EPSC) platform modeling to STAT-1 GoF, we identified some potential candidates, which not only inhibit STAT-1 function independently but also synergize with existing clinical treatments. Our findings suggest a promising avenue for clinical applications, offering new insights for personalized therapeutic strategies in IEI management
Funding Source: Funding support from Health@InnoHK, Innovation and Technology Commission
