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Emerging Therapies for Childhood Polycystic Kidney Disease
#MMPMID28473970
Sweeney WE Jr
; Avner ED
Front Pediatr
2017[]; 5
(?): 77
PMID28473970
show ga
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.
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