نتایج جستجو برای: ciliopathy

تعداد نتایج: 423  

Journal: :Human molecular genetics 2013
Monique N H Luijten Sander G Basten Tijs Claessens Marigje Vernooij Claire L Scott Renske Janssen Jennifer A Easton Miriam A F Kamps Maaike Vreeburg Jos L V Broers Michel van Geel Fred H Menko Richard P Harbottle Ravi K Nookala Andrew R Tee Stephen C Land Rachel H Giles Barry J Coull Maurice A M van Steensel

Birt-Hogg-Dubé (BHD) syndrome is an autosomal dominant disorder where patients are predisposed to kidney cancer, lung and kidney cysts and benign skin tumors. BHD is caused by heterozygous mutations affecting folliculin (FLCN), a conserved protein that is considered a tumor suppressor. Previous research has uncovered multiple roles for FLCN in cellular physiology, yet it remains unclear how the...

Journal: :The Journal of clinical investigation 2009
Norann A Zaghloul Nicholas Katsanis

Bardet-Biedl syndrome (BBS) is a multisystemic disorder typified by developmental and progressive degenerative defects. A combination of genetic, in vitro, and in vivo studies have highlighted ciliary dysfunction as a primary cause of BBS pathology, which has in turn contributed to the improved understanding of the functions of the primary cilium in humans and other vertebrates. Here we discuss...

Journal: :Human mutation 2014
Bodo B Beck Jennifer B Phillips Malte P Bartram Jeremy Wegner Michaela Thoenes Andrea Pannes Josephina Sampson Raoul Heller Heike Göbel Friederike Koerber Antje Neugebauer Andrea Hedergott Gudrun Nürnberg Peter Nürnberg Holger Thiele Janine Altmüller Mohammad R Toliat Simon Staubach Kym M Boycott Enza Maria Valente Andreas R Janecke Tobias Eisenberger Carsten Bergmann Lars Tebbe Yang Wang Yundong Wu Andrew M Fry Monte Westerfield Uwe Wolfrum Hanno J Bolz

We describe a consanguineous Iraqi family with Leber congenital amaurosis (LCA), Joubert syndrome (JBTS), and polycystic kidney disease (PKD). Targeted next-generation sequencing for excluding mutations in known LCA and JBTS genes, homozygosity mapping, and whole-exome sequencing identified a homozygous missense variant, c.317G>C (p.Arg106Pro), in POC1B, a gene essential for ciliogenesis, basal...

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