Engineered Alzheimer Organoids Validate the Link Between Intracellular and Soluble p-Tau Biomarkers and Highlight the Contribution of Astrocytic Tau

  • Neurosci Bull. 2026 Apr 11. doi: 10.1007/s12264-026-01615-4.
Ru Zhang  1  2 Xiaoxu Dong  3 Gang Pei  1  4  5  2 Shichao Huang  6  7
Affiliations
  • 1. State Key Laboratory of Molecular Biology, Shanghai Institute of Biochemistry and Cell Biology, Center for Excellence in Molecular Cell Science, Chinese Academy of Sciences, Shanghai, 200031, China.
  • 2. University of Chinese Academy of Sciences, Beijing, 101408, China.
  • 3. Department of Colorectal Surgery, Fudan University Shanghai Cancer Center, Shanghai, 200032, China.
  • 4. Shanghai Key Laboratory of Signaling and Disease Research, Laboratory of Receptor-based Biomedicine, The Collaborative Innovation Center for Brain Science, School of Life Sciences and Technology, Tongji University, Shanghai, 200092, China.
  • 5. Institute for Stem Cell and Regeneration, Chinese Academy of Sciences, Beijing, 100101, China.
  • 6. State Key Laboratory of Molecular Biology, Shanghai Institute of Biochemistry and Cell Biology, Center for Excellence in Molecular Cell Science, Chinese Academy of Sciences, Shanghai, 200031, China. [email protected].
  • 7. University of Chinese Academy of Sciences, Beijing, 101408, China. [email protected].
Abstract

Soluble phosphorylated tau has become a key biomarker for Alzheimer's disease pathology, yet further mechanistic studies are needed beyond observational investigations to clarify the relationship between soluble p-tau species and intracellular tau pathology. Here, we utilized chimeric human cerebral organoids (chCOs) to generate a series of organoids in which the endogenous MAPT gene was CRISPR/Cas9 edited in different cell types to create phosphorylation-deficient mutants at the AD-associated sites. We found that the APPswe mutation increased tau phosphorylation in both neurons and astrocytes. Notably, astrocyte-specific phosphorylation-deficient mutations of tau in organoids reduced soluble p-tau181 and p-tau217 levels, as detected by single-molecule array. These findings indicate that astrocytic tau plays a substantial role in contributing to the pool of extracellular phosphorylated tau and suggest it may be an overlooked source of AD biomarkers. Moreover, our engineered chCOs offer a versatile platform for exploring how cell-type-specific pathologies correlate with changes in biomarker profiles.

Keywords
Alzheimer’s disease; Astrocytes; Human chimeric brain organoids; Tau pathology; p-Tau biomarkers.
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