Ciliopathies

F Hildebrandt, T Benzing… - New England Journal of …, 2011 - Mass Medical Soc
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Ciliopathies: an expanding disease spectrum

AM Waters, PL Beales - Pediatric nephrology, 2011 - Springer
Ciliopathies comprise a group of disorders associated with genetic mutations encoding
defective proteins, which result in either abnormal formation or function of cilia. As cilia are a …

Renal plasticity revealed through reversal of polycystic kidney disease in mice

K Dong, C Zhang, X Tian, D Coman, F Hyder, M Ma… - Nature …, 2021 - nature.com
Initiation of cyst formation in autosomal dominant polycystic kidney disease (ADPKD) occurs
when kidney tubule cells are rendered null for either PKD1 or PKD2 by somatic 'second …

Autosomal dominant polycystic kidney disease

VE Torres, PC Harris, Y Pirson - The Lancet, 2007 - thelancet.com
Autosomal dominant polycystic kidney disease is the most prevalent, potentially lethal,
monogenic disorder. It is associated with large interfamilial and intrafamilial variability, which …

Genetic mechanisms and signaling pathways in autosomal dominant polycystic kidney disease

PC Harris, VE Torres - The Journal of clinical investigation, 2014 - Am Soc Clin Investig
Recent advances in defining the genetic mechanisms of disease causation and modification
in autosomal dominant polycystic kidney disease (ADPKD) have helped to explain some …

Functional polycystin-1 dosage governs autosomal dominant polycystic kidney disease severity

K Hopp, CJ Ward, CJ Hommerding… - The Journal of …, 2012 - Am Soc Clin Investig
Autosomal dominant polycystic kidney disease (ADPKD) is caused by mutations to PKD1 or
PKD2, triggering progressive cystogenesis and typically leading to end-stage renal disease …

Polycystic kidney disease

PC Harris, VE Torres - Annual review of medicine, 2009 - annualreviews.org
A number of inherited disorders result in renal cyst development. The most common form,
autosomal dominant polycystic kidney disease (ADPKD), is a disorder most often diagnosed …

Loss of cilia suppresses cyst growth in genetic models of autosomal dominant polycystic kidney disease

M Ma, X Tian, P Igarashi, GJ Pazour, S Somlo - Nature genetics, 2013 - nature.com
Kidney cysts occur following inactivation of polycystins in otherwise intact cilia or following
complete removal of cilia by inactivation of intraflagellar transport–related proteins. We …

[HTML][HTML] Autosomal dominant polycystic kidney disease: the last 3 years

VE Torres, PC Harris - Kidney international, 2009 - Elsevier
Autosomal dominant polycystic kidney disease is the most prevalent, potentially lethal
monogenic disorder. It has large inter-and intra-familial variability explained to a large extent …

[HTML][HTML] Exome capture reveals ZNF423 and CEP164 mutations, linking renal ciliopathies to DNA damage response signaling

M Chaki, R Airik, AK Ghosh, RH Giles, R Chen… - Cell, 2012 - cell.com
Nephronophthisis-related ciliopathies (NPHP-RC) are degenerative recessive diseases that
affect kidney, retina, and brain. Genetic defects in NPHP gene products that localize to cilia …