emerging therapies for childhood polycystic kidney disease

Clicks: 239
ID: 198444
2017
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Abstract
Cystic kidney diseases comprise a varied collection of hereditary disorders, where renal cysts comprise a major element of their pleiotropic phenotype. In pediatric patients, the term polycystic kidney disease (PKD) commonly refers to two specific hereditary diseases, autosomal recessive polycystic kidney disease (ARPKD) and autosomal dominant polycystic kidney disease (ADPKD). Remarkable progress has been made in understanding the complex molecular and cellular mechanisms of renal cyst formation in ARPKD and ADPKD. One of the most important discoveries is that both the genes and proteins products of ARPKD and ADPKD interact in a complex network of genetic and functional interactions. These interactions and the shared phenotypic abnormalities of ARPKD and ADPKD, the “cystic phenotypes” suggest that many of the therapies developed and tested for ADPKD may be effective in ARPKD as well. Successful therapeutic interventions for childhood PKD will, therefore, be guided by knowledge of these molecular interactions, as well as a number of clinical parameters, such as the stage of the disease and the rate of disease progression.
Reference Key
sweeney2017frontiersemerging Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors ;William E. Sweeney;Ellis D. Avner
Journal disease markers
Year 2017
DOI
10.3389/fped.2017.00077
URL
http://journal.frontiersin.org/article/10.3389/fped.2017.00077/full
Keywords
therapy combination therapy

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