Modeling Pkd1 gene-targeted strategies for correction of polycystic kidney disease

Modeling Pkd1 gene-targeted strategies for correction of polycystic kidney disease

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Autosomal dominant polycystic kidney disease (ADPKD) causes renal cysts and leads to end-stage renal disease in midlife due mainly to PKD1 gene mutations. Virtually no studies have explored gene therapeutic strategies for long-term effective treatment of PKD. Toward this aim, the severely cystic Pkd...

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Bibliographic Details
Main Authors:	Almira Kurbegovic, Rey Christian Pacis, Marie Trudel
Format:	Article
Language:	English
Published:	Elsevier 2023-06-01
Series:	Molecular Therapy: Methods & Clinical Development
Subjects:	Pkd1 gene therapy Pkd1 locus/minigene polycystic kidney disease PKD transcriptional regulation small SB renal cassette Pkd1 RNAscope
Online Access:	http://www.sciencedirect.com/science/article/pii/S2329050123000529

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