TY - JOUR
T1 - MicroRNAs and polycystic kidney disease
AU - Noureddine, Lama
AU - Hajarnis, Sachin
AU - Patel, Vishal
N1 - Funding Information:
The work in our laboratory is supported by grants from the National Institutes of Health/National Institute of Diabetes and Digestive and Kidney Diseases ( KO8 DK084311-01 and RO3DK099568-01 to VP), the PKD foundation (to VP) and the UT Southwestern O’Brien Kidney Research Core Center ( P30 DK079328 ). LN is supported by a T32 training grant ( DK007257 ) from NIDDK .
PY - 2013
Y1 - 2013
N2 - Polycystic kidney disease (PKD), the most common genetic cause of chronic renal failure, is characterized by the presence of numerous fluid-filled cysts in renal parenchyma. Despite recent progress, no FDA-approved therapy is available to retard cyst growth. Here, we review current evidence implicating two groups of microRNAs (miRNAs) - the miR-17∼92 cluster and miR-200s - in the pathogenesis of PKD. We present a new hypothesis for cyst growth involving miRNAs and regulation of PKD gene dosage. We propose that manipulating miRNA function in an attempt to normalize PKD gene dosage represents a novel therapeutic strategy in PKD.
AB - Polycystic kidney disease (PKD), the most common genetic cause of chronic renal failure, is characterized by the presence of numerous fluid-filled cysts in renal parenchyma. Despite recent progress, no FDA-approved therapy is available to retard cyst growth. Here, we review current evidence implicating two groups of microRNAs (miRNAs) - the miR-17∼92 cluster and miR-200s - in the pathogenesis of PKD. We present a new hypothesis for cyst growth involving miRNAs and regulation of PKD gene dosage. We propose that manipulating miRNA function in an attempt to normalize PKD gene dosage represents a novel therapeutic strategy in PKD.
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U2 - 10.1016/j.ddmod.2013.10.001
DO - 10.1016/j.ddmod.2013.10.001
M3 - Review article
C2 - 25221607
AN - SCOPUS:84904061085
SN - 1740-6757
VL - 10
SP - e137-e143
JO - Drug Discovery Today: Disease Models
JF - Drug Discovery Today: Disease Models
IS - 3
ER -