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September 19, 2025BrainOpen Access

PU.1 restores microglial dysfunction caused by C9ORF72 repeat expansions in neural organoids

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Authors

TLTijana LjubikjMMMayte MarsAGAstrid T. van der Geest

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Overview

Observational analysis reveals impaired microglial function in neural organoids from ALS patients, suggesting PU.1 could restore these changes.

Key Points

  • Reduced phagocytosis in microglia derived from neural organoids of C9-ALS patients shows significant functional impairment.
  • Transcriptomics unveiled PU.1 as the most downregulated transcription factor in C9-oMGs, highlighting its potential therapeutic role.
  • The study utilized cerebral organoids from iPSCs of C9-ALS/FTD patients to model microglial dysfunction in a complex environment.
  • Findings indicate that PU.1 overexpression can restore microglial functions, potentially guiding future ALS therapeutic strategies.

Cite This Study

Ljubikj et al. (2025) studied this question.

synapsesocial.com/papers/68d464ea31b076d99fa63ecfhttps://doi.org/10.1093/brain/awaf340
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  4. 4Moderate intrinsic phenotypic alterations in C9orf72 ALS/FTD iPSC-microglia despite the presence of C9orf72 pathological features2023 · 24 citations
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