Novel therapeutic approaches to autosomal dominant polycystic kidney disease

Research output: Contribution to journalReview articlepeer-review

17 Scopus citations

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is an inherited disorder characterized by the progressive growth of renal cysts that, over time, destroy the architecture of the renal parenchyma and typically lead to kidney failure by the sixth decade of life. ADPKD is common and represents a leading cause of renal failure worldwide. Currently, there are no Food and Drug Administration-approved treatments for the disease, and the existing standard of care is primarily supportive in nature. However, significant advances in the understanding of the molecular biology of the disease have inspired investigation into potential new therapies. Several drugs designed to slow or arrest the progression of ADPKD have shown promise in preclinical models and clinical trials, including vasopressin receptor antagonists and somatostatin analogs. This article examines the literature underlying the rationale for molecular therapies for ADPKD and reviews the existing clinical evidence for their indication for human patients with the disease.

Original languageEnglish (US)
Article number852
Pages (from-to)488-498
Number of pages11
JournalTranslational Research
Volume165
Issue number4
DOIs
StatePublished - Apr 1 2015

Keywords

  • ADPKD
  • AMP-activated protein kinase
  • AMPK
  • CFTR
  • ESRD
  • PC1
  • PC2
  • V2R
  • autosomal dominant polycystic kidney disease
  • cAMP
  • cyclic adenosine 3',5'-monophosphate
  • cystic fibrosis transmembrane conductance regulator
  • end-stage renal disease
  • mTOR
  • mammalian target of rapamycin
  • polycystin-1
  • polycystin-2
  • vasopressin V2 receptor

ASJC Scopus subject areas

  • Public Health, Environmental and Occupational Health
  • Biochemistry, medical
  • Physiology (medical)

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