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Background <p>Neurodegenerative disorders, including Alzheimer's disease (AD) and Parkinson's disease (PD), remain major global health challenges owing to their progressive pathology, limited regenerative capacity of the central nervous system, and lack of effective disease-modifying therapies. Conventional in vitro and animal models often fail to accurately reproduce the complexity of the human brain, limiting translational success. Brain organoids have emerged as advanced three-dimensional (3D) human stem cell-derived models that closely mimic the structural and functional characteristics of the developing and diseased brain, providing a promising platform for investigating disease mechanisms and therapeutic interventions.</p> Method <p>This review comprehensively summarizes recent advances in brain organoid technology and its applications in modelling AD and PD. Relevant studies were critically evaluated to examine organoid generation strategies, disease modelling approaches, pathological features, patient-derived organoid applications, drug screening, toxicity assessment, biomarker discovery, and emerging technologies, including microfluidic integration, genome editing, and artificial intelligence, with emphasis on their translational relevance.</p> Results <p>Brain organoids successfully recapitulate key pathological hallmarks of AD and PD, including amyloid-β accumulation, hyperphosphorylated tau pathology, α-synuclein aggregation, neuronal degeneration, and neuroinflammatory responses. Patient-specific organoids effectively capture disease heterogeneity and facilitate precision medicine approaches through individualized drug screening and therapeutic evaluation. Furthermore, integration with organ-on-chip systems, CRISPR-based genome editing, and artificial intelligence enhances disease modelling, functional analysis, and predictive drug discovery. Despite challenges related to reproducibility, vascularization, cellular maturation, scalability, and regulatory standardization, brain organoids demonstrate superior physiological relevance compared with conventional experimental models.</p> Conclusion <p>Brain organoids represent a transformative platform for studying the pathogenesis of neurodegenerative diseases and accelerating translational neuroscience. Their ability to model patient-specific disease mechanisms and support personalized therapeutic development positions them as valuable tools for improving drug discovery, biomarker identification, and precision medicine. Continued technological refinement and standardized protocols are expected to further expand their clinical and research applications in AD, PD, and other neurodegenerative disorders.</p>

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Brain Organoids in Alzheimer’s and Parkinson’s Disease: Clinical Applications and Translational Perspectives

  • 期刊论文

  • 发表日期：

    2026年7月31日

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  • Prathiba Balaji,
  • Nandhini Jayaprakash

摘要

Background

Neurodegenerative disorders, including Alzheimer's disease (AD) and Parkinson's disease (PD), remain major global health challenges owing to their progressive pathology, limited regenerative capacity of the central nervous system, and lack of effective disease-modifying therapies. Conventional in vitro and animal models often fail to accurately reproduce the complexity of the human brain, limiting translational success. Brain organoids have emerged as advanced three-dimensional (3D) human stem cell-derived models that closely mimic the structural and functional characteristics of the developing and diseased brain, providing a promising platform for investigating disease mechanisms and therapeutic interventions.

Method

This review comprehensively summarizes recent advances in brain organoid technology and its applications in modelling AD and PD. Relevant studies were critically evaluated to examine organoid generation strategies, disease modelling approaches, pathological features, patient-derived organoid applications, drug screening, toxicity assessment, biomarker discovery, and emerging technologies, including microfluidic integration, genome editing, and artificial intelligence, with emphasis on their translational relevance.

Results

Brain organoids successfully recapitulate key pathological hallmarks of AD and PD, including amyloid-β accumulation, hyperphosphorylated tau pathology, α-synuclein aggregation, neuronal degeneration, and neuroinflammatory responses. Patient-specific organoids effectively capture disease heterogeneity and facilitate precision medicine approaches through individualized drug screening and therapeutic evaluation. Furthermore, integration with organ-on-chip systems, CRISPR-based genome editing, and artificial intelligence enhances disease modelling, functional analysis, and predictive drug discovery. Despite challenges related to reproducibility, vascularization, cellular maturation, scalability, and regulatory standardization, brain organoids demonstrate superior physiological relevance compared with conventional experimental models.

Conclusion

Brain organoids represent a transformative platform for studying the pathogenesis of neurodegenerative diseases and accelerating translational neuroscience. Their ability to model patient-specific disease mechanisms and support personalized therapeutic development positions them as valuable tools for improving drug discovery, biomarker identification, and precision medicine. Continued technological refinement and standardized protocols are expected to further expand their clinical and research applications in AD, PD, and other neurodegenerative disorders.