Renal Resilience - Recent Advances in Clinical Knowledge and Future Treatment of Rare Orphan Diseases; ISN-CSN Joint Session
Session description:
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.
Learning objectives:
Review renal complications and therapeutic approaches of sickle cell disease. Learn the perspectives of patient derived induced pluripotent stem cells to model genetic kidney diseases. Learn advancements of gene therapy for rare genetic diseases.
| Time | Session |
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9:45 a.m.
10:03 a.m.
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How Do I Treat Renal Complications in Patients With Sickle Cell Disease?
ClaireSharpe
Speaker
claire.sharpe@nottingham.ac.ukUniversity of Nottingham and King's College Hospital, LondonUnited Kingdom
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10:03 a.m.
10:21 a.m.
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iPSCs to Model Rare Kidney Diseases for Drug Discovery: The Examples of FSGS and ADPKD
ArielaBenigni
Speaker
ariela.benigni@marionegri.itMario Negri Institute for Pharmacological Research, ItalyItaly
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10:21 a.m.
10:39 a.m.
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Fabry's Disease: Clinical Features and Novel Therapeutics
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10:39 a.m.
10:57 a.m.
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Primary Hyperoxaluria: A Hope for Gene Therapy?
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10:57 a.m.
11:15 a.m.
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Q&A
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