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

ABSTRACT: 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 Pkd1-null mouse model was targeted with a series of transgene transfers using genomic Pkd1 under its regulatory elements (Pkd1^wt), a kidney-targeted Pkd1 gene (^SBPkd1), or Pkd1^Minigene. The introduced Pkd1^wt gene constructs with ∼8-fold overexpression display similar endogenous cellular profiles and full complementation of Pkd1^-/- phenotype and establish

SUBMITTER: Kurbegovic A 

PROVIDER: S-EPMC10199405 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature