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Autosomal Dominant Polycystic Kidney Disease: From Pathogenesis to Organoid Disease Models
Alexandru Scarlat1, Susanna Tomasoni1, Piera Trionfini1
1Istituto di Ricerche Farmacologiche Mario Negri IRCCS, Centro Anna Maria Astori, Science and Technology Park Kilometro Rosso, 24126 Bergamo, Italy.
Insights
Pluripotent stem cell-derived kidney organoids offer a human-relevant model for studying autosomal dominant polycystic kidney disease (ADPKD). These models advance understanding of ADPKD pathogenesis and aid in screening potential therapies.
Area of Science:
- Nephrology and Regenerative Medicine
- Genetics and Molecular Biology
Background:
- Autosomal dominant polycystic kidney disease (ADPKD), caused by mutations in PKD1 or PKD2, is the leading inherited renal disorder.
- Current ADPKD therapies lack efficacy in halting disease progression, highlighting the need for improved human-relevant models.
- Preclinical animal models show limited translatability to human ADPKD.
Purpose of the Study:
- To review the development and application of pluripotent stem cell (PSC)-derived kidney organoids as models for ADPKD.
- To discuss how these organoids enhance understanding of ADPKD pathogenesis.
- To explore the limitations of current PSC-derived kidney organoid models and propose strategies for improvement.
Main Methods:
- Utilizing pluripotent stem cells (PSCs) to generate kidney organoids that mimic human renal architecture and function.
- Employing PSC-derived kidney organoids to study ADPKD pathogenesis.
- Screening potential therapeutic drug candidates using these advanced in vitro models.
Main Results:
- PSC-derived kidney organoids provide a physiologically relevant platform for ADPKD research.
- These models have identified numerous dysregulated pathways implicated in ADPKD.
- Drug screening in organoids has shown promise for identifying effective ADPKD therapies.
Conclusions:
- PSC-derived kidney organoids represent a significant advancement in modeling ADPKD.
- These models are crucial for dissecting disease mechanisms and accelerating therapeutic development.
- Further refinement of organoid models will enhance their utility for clinical applications in ADPKD.
Abstract:
Mutations in PKD1 and PKD2 cause autosomal dominant polycystic kidney disease (ADPKD), the most common renal genetic disease, leading to the dysregulation of renal tubules and the development of cystic growth that compromises kidney function. Despite significant advances in recent decades, there remains a considerable unmet clinical need, as current therapeutics are not effective at slowing or halting disease progression. Although preclinical animal models have been used extensively, the translatability of such findings is uncertain and human-relevant disease models are urgently needed. The advent of pluripotent stem cells (PSCs) and their ability to more accurately recapitulate organ architecture and function has allowed for the study of renal disease in a more physiological and human-relevant setting. To date, many research groups have studied ADPKD using PSC-derived kidney organoids, identifying many dysregulated pathways and screening drug candidates that may yield effective therapies in the clinic. In this review article, we discuss in detail the development of PSC-derived kidney organoids as ADPKD models and how they have advanced our understanding of the disease's pathogenesis, as well as their limitations and potential strategies to address them.
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