Literature DB >> 22910529

Severe aortic stenosis, bicuspid aortic valve and atrial septal defect in a child with Joubert Syndrome and Related Disorders (JSRD) - a case report and review of congenital heart defects reported in the human ciliopathies.

Natalya Karp1, Lars Grosse-Wortmann, Sarah Bowdin.   

Abstract

We report a case of a 2 year-old boy with Joubert Syndrome and Related Disorders (JSRD), severe congenital aortic stenosis, bicuspid aortic valve and an atrial septal defect. JSRD is one of a group of conditions known as 'ciliopathies', whose multi-organ involvement results from primary cilia dysfunction. To date, there have been no other reported cases of aortic stenosis and bicuspid aortic valve associated with JSRD. Cardiac screening is not currently recommended in the management guidelines for individuals suspected of having JSRD. We speculate that while the presence of congenital aortic stenosis in this child could be caused by an unrelated genetic mechanism, it could also represent a phenotypic overlap with another ciliopathy, Bardet Biedl syndrome, in which aortic stenosis is more commonly reported. We also review the range of cardiac malformations reported to be present in all human diseases known to be ciliopathies, in order to assist with the investigation and management of individuals with a suspected or proven ciliopathy.
Copyright © 2012 Elsevier Masson SAS. All rights reserved.

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Year:  2012        PMID: 22910529     DOI: 10.1016/j.ejmg.2012.07.010

Source DB:  PubMed          Journal:  Eur J Med Genet        ISSN: 1769-7212            Impact factor:   2.708


  17 in total

Review 1.  Cilia and coordination of signaling networks during heart development.

Authors:  Karen Koefoed; Iben Rønn Veland; Lotte Bang Pedersen; Lars Allan Larsen; Søren Tvorup Christensen
Journal:  Organogenesis       Date:  2013-12-17       Impact factor: 2.500

2.  Nystagmus in a newborn: a manifestation of Joubert syndrome in the neonatal period.

Authors:  Inês Salva; Carolina Albuquerque; Ana Moreira; Catarina Dâmaso
Journal:  BMJ Case Rep       Date:  2016-01-12

3.  Ruptured Sinus of Valsalva Aneurysm Associated with Situs Ambiguus, Isolated Levocardia, and Polysplenia.

Authors:  Safieh Golestaneh; Mohammad Amin Kashef; William L Hiser; Amir S Lotfi; Timothy G Egan
Journal:  Tex Heart Inst J       Date:  2017-12-19

Review 4.  Healthcare recommendations for Joubert syndrome.

Authors:  Ruxandra Bachmann-Gagescu; Jennifer C Dempsey; Sara Bulgheroni; Maida L Chen; Stefano D'Arrigo; Ian A Glass; Theo Heller; Elise Héon; Friedhelm Hildebrandt; Nirmal Joshi; Dana Knutzen; Hester Y Kroes; Stephen H Mack; Sara Nuovo; Melissa A Parisi; Joseph Snow; Angela C Summers; Jordan M Symons; Wadih M Zein; Eugen Boltshauser; John A Sayer; Meral Gunay-Aygun; Enza Maria Valente; Dan Doherty
Journal:  Am J Med Genet A       Date:  2019-11-11       Impact factor: 2.802

5.  A role for primary cilia in aortic valve development and disease.

Authors:  Katelynn A Toomer; Diana Fulmer; Lilong Guo; Alex Drohan; Neal Peterson; Paige Swanson; Brittany Brooks; Rupak Mukherjee; Simon Body; Joshua H Lipschutz; Andy Wessels; Russell A Norris
Journal:  Dev Dyn       Date:  2017-06-28       Impact factor: 3.780

Review 6.  Hedgehog Morphogens Act as Growth Factors Critical to Pre- and Postnatal Cardiac Development and Maturation: How Primary Cilia Mediate Their Signal Transduction.

Authors:  Lindsey A Fitzsimons; Victoria L Brewer; Kerry L Tucker
Journal:  Cells       Date:  2022-06-09       Impact factor: 7.666

7.  Cilia gene mutations cause atrioventricular septal defects by multiple mechanisms.

Authors:  Ozanna Burnicka-Turek; Jeffrey D Steimle; Wenhui Huang; Lindsay Felker; Anna Kamp; Junghun Kweon; Michael Peterson; Roger H Reeves; Cheryl L Maslen; Peter J Gruber; Xinan H Yang; Jay Shendure; Ivan P Moskowitz
Journal:  Hum Mol Genet       Date:  2016-06-23       Impact factor: 6.150

8.  Desert hedgehog-primary cilia cross talk shapes mitral valve tissue by organizing smooth muscle actin.

Authors:  Diana Fulmer; Katelynn A Toomer; Janiece Glover; Lilong Guo; Kelsey Moore; Reece Moore; Rebecca Stairley; Cortney Gensemer; Sameer Abrol; Mary Kate Rumph; Faith Emetu; Joshua H Lipschutz; Colin McDowell; Justin Bian; Christina Wang; Tyler Beck; Andy Wessels; Marie-Ange Renault; Russell A Norris
Journal:  Dev Biol       Date:  2020-03-06       Impact factor: 3.582

9.  Defects in the Exocyst-Cilia Machinery Cause Bicuspid Aortic Valve Disease and Aortic Stenosis.

Authors:  Diana Fulmer; Katelynn Toomer; Lilong Guo; Kelsey Moore; Janiece Glover; Reece Moore; Rebecca Stairley; Glenn Lobo; Xiaofeng Zuo; Yujing Dang; Yanhui Su; Ben Fogelgren; Patrick Gerard; Dongjun Chung; Mahyar Heydarpour; Rupak Mukherjee; Simon C Body; Russell A Norris; Joshua H Lipschutz
Journal:  Circulation       Date:  2019-08-07       Impact factor: 29.690

Review 10.  Role of cilia in the pathogenesis of congenital heart disease.

Authors:  George C Gabriel; Cullen B Young; Cecilia W Lo
Journal:  Semin Cell Dev Biol       Date:  2020-05-14       Impact factor: 7.727

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