Article
Envisioning treating genetically-defined urinary tract malformations with viral vector-mediated gene therapy
7 Jul 2021
Abstract excerpt
Human urinary tract malformations can cause dysfunctional voiding, urosepsis and kidney failure. Other affected individuals, with severe phenotypes on fetal ultrasound screening, undergo elective termination. Currently, there exist no specific treatments that target the primary biological disease mechanisms that generate these urinary tract malformations. Historically, the pathogenesis of human urinary tract...
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