Genetic basis for congenital heart disease: revisited: a scientific statement from the American Heart Association

ME Pierpont, M Brueckner, WK Chung, V Garg… - Circulation, 2018 - Am Heart Assoc
This review provides an updated summary of the state of our knowledge of the genetic
contributions to the pathogenesis of congenital heart disease. Since 2007, when the initial …

Ciliary disorder of the skeleton

C Huber, V Cormier‐Daire - … Journal of Medical Genetics Part C …, 2012 - Wiley Online Library
In the last 10 years, the primary cilia machinery has been implicated in more than a dozen
disorders united as ciliopathies, including skeletal dysplasias, such as Jeune syndrome and …

Genome-wide analyses identify KIF5A as a novel ALS gene

A Nicolas, KP Kenna, AE Renton, N Ticozzi, F Faghri… - Neuron, 2018 - cell.com
To identify novel genes associated with ALS, we undertook two lines of investigation. We
carried out a genome-wide association study comparing 20,806 ALS cases and 59,804 …

Ellis-van Creveld syndrome

G Baujat, M Le Merrer - Orphanet journal of rare diseases, 2007 - Springer
Ellis-van Creveld syndrome (EVC) is a chondral and ectodermal dysplasia characterized by
short ribs, polydactyly, growth retardation, and ectodermal and heart defects. It is a rare …

The retinal ciliopathies

NA Adams, A Awadein, HS Toma - Ophthalmic genetics, 2007 - Taylor & Francis
While the functions of many of the proteins located in or associated with the photoreceptor
cilia are poorly understood, disruption of the function of these proteins may result in a wide …

NEK1 mutations cause short-rib polydactyly syndrome type majewski

C Thiel, K Kessler, A Giessl, A Dimmler… - The American Journal of …, 2011 - cell.com
Defects of ciliogenesis have been implicated in a wide range of human phenotypes and play
a crucial role in signal transduction and cell-cycle coordination. We used homozygosity …

Evc is a positive mediator of Ihh-regulated bone growth that localises at the base of chondrocyte cilia

VL Ruiz-Perez, HJ Blair, ME Rodriguez-Andres… - 2007 - journals.biologists.com
EVC is a novel protein mutated in the human chondroectodermal dysplasia Ellis-van
Creveld syndrome (EvC; OMIM: 225500). We have inactivated Evc in the mouse and show …

The pathogenesis of atrial and atrioventricular septal defects with special emphasis on the role of the dorsal mesenchymal protrusion

LE Briggs, J Kakarla, A Wessels - Differentiation, 2012 - Elsevier
Partitioning of the four-chambered heart requires the proper formation, interaction and fusion
of several mesenchymal tissues derived from different precursor populations that together …

Function and regulation of primary cilia and intraflagellar transport proteins in the skeleton

X Yuan, RA Serra, S Yang - Annals of the New York Academy …, 2015 - Wiley Online Library
Primary cilia are microtubule‐based organelles that project from the cell surface to enable
transduction of various developmental signaling pathways. The process of intraflagellar …

Skeletal ciliopathies: a pattern recognition approach

A Handa, U Voss, A Hammarsjö… - Japanese journal of …, 2020 - Springer
Ciliopathy encompasses a diverse group of autosomal recessive genetic disorders caused
by mutations in genes coding for components of the primary cilia. Skeletal ciliopathy forms a …