WCN'25 Renal Resilience - Recent Advances in Clinical Knowledge and Future Treatment of Rare Orphan Diseases; ISN-CSN Joint Session

Only available as part of a package

This session will highlight the clinical issues and emerging treatments of prototypical genetic diseases, which either directly target the kidney or cause severe renal complications. How to manage patients with sickle-related renal complications will be discussed. The advancements in the use of induced pluripotent stem cells from patients to model genetic focal segmental glomerulosclerosis and autosomal dominant polycystic kidney disease will be presented. Finally, novel therapeutic approaches for Fabry’s diseases and primary hyperoxaluria, including gene therapy with viral and non-viral vectors will be described.

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How Do I Treat Renal Complications in Patients With Sickle Cell Disease?
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iPSCs to Model Rare Kidney Diseases for Drug Discovery: The Examples of FSGS and ADPKD
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Open to view video.
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Kidney Multiome-based Genetic Scorecard Reveals Convergent Coding and Regulatory Variants
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Open to view video.
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Primary Hyperoxaluria: A Hope for Gene Therapy?
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Open to view video.
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