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Ciliopathy organoid models: a comprehensive review.
Matylda Zofia Kuzinska1,2, Sally Yuan-Yin Lin1, Verena Klämbt1,3
1Department of Pediatric Gastroenterology, Nephrology and Metabolic Diseases, Charité Universitätsmedizin Berlin-Campus Virchow Klinikum, Berlin, Germany.
American Journal of Physiology. Cell Physiology
|November 4, 2024
Summary
Organoids derived from human cells offer a powerful new way to study ciliopathies, a group of genetic disorders affecting cilia. These models help researchers understand disease mechanisms and test potential treatments for conditions like polycystic kidney disease.
Area of Science:
- Cell Biology
- Genetics
- Developmental Biology
Background:
- Cilia are vital organelles involved in sensing and signaling, crucial for human physiology and development.
- Ciliopathies, genetic disorders of cilia, have a high collective prevalence despite individual rarity, impacting organs like the kidney, airway, liver, and retina.
- Existing animal models have limitations in replicating human ciliopathy's phenotypic heterogeneity.
Purpose of the Study:
- To review and evaluate current organoid models for studying ciliopathies.
- To highlight organoid applications in understanding patient-specific genotype-phenotype correlations.
- To summarize methodologies for cultivating and analyzing ciliary dysfunction in organoids.
Main Methods:
- Utilizing self-assembled three-dimensional organoids derived from human induced pluripotent stem cells or primary tissues.
- Focusing on kidney, airway, liver, and retinal organoid models.
- Describing cultivation techniques and methods for interrogating ciliary dysfunction.
Main Results:
- Organoids recapitulate organ-specific development, architecture, and function.
- Organoid models show promise for patient-specific disease modeling and drug screening.
- The review synthesizes current knowledge on various organoid types for ciliopathy research.
Conclusions:
- Organoids represent a significant advancement in ciliopathy research, offering better human-relevant models.
- These models facilitate the study of genotype-phenotype correlations and preclinical drug development.
- Organoid technology is poised to accelerate therapeutic discoveries for ciliopathies.
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