Literature DB >> 2929661

Further delineation of a syndrome of cerebellar vermis hypo/aplasia, oligophrenia, congenital ataxia, coloboma, and hepatic fibrosis.

A Verloes1, C Lambotte.   

Abstract

Three children are described from two sibships. They share infantile ataxia with hypo/aplastic vermis, hepatic fibrocirrhosis, slender-shaped skeleton, peculiar face, and moderate mental retardation. One of them had a kidney biopsy that showed mild interstitial fibrosis and amyloid deposit, but had no functional impairment. Another suffered moderate proximal tubular acidosis. Two children had unilateral or bilateral choroidal coloboma. This pattern of defects is consistent with a syndrome previously reported in two other sibships. The acronym COACH (Cerebellar vermis hypo/aplasia, Oligophrenia, congenital Ataxia, Coloboma, Hepatic fibrocirrhosis) is suggested.

Entities:  

Mesh:

Year:  1989        PMID: 2929661     DOI: 10.1002/ajmg.1320320217

Source DB:  PubMed          Journal:  Am J Med Genet        ISSN: 0148-7299


  18 in total

Review 1.  Joubert Syndrome and related disorders.

Authors:  Francesco Brancati; Bruno Dallapiccola; Enza Maria Valente
Journal:  Orphanet J Rare Dis       Date:  2010-07-08       Impact factor: 4.123

Review 2.  Clinical and molecular features of Joubert syndrome and related disorders.

Authors:  Melissa A Parisi
Journal:  Am J Med Genet C Semin Med Genet       Date:  2009-11-15       Impact factor: 3.908

3.  Dealing with congenital hepatic fibrosis? Remember COACH syndrome.

Authors:  B C Acharyya; M K Goenka; S Chatterjee; U Goenka
Journal:  Clin J Gastroenterol       Date:  2013-12-11

Review 4.  Ocular coloboma: a reassessment in the age of molecular neuroscience.

Authors:  C Y Gregory-Evans; M J Williams; S Halford; K Gregory-Evans
Journal:  J Med Genet       Date:  2004-12       Impact factor: 6.318

5.  Further delineation of renal-coloboma syndrome in patients with extreme variability of phenotype and identical PAX2 mutations.

Authors:  L A Schimmenti; H E Cunliffe; L A McNoe; T A Ward; M C French; H H Shim; Y H Zhang; W Proesmans; A Leys; K A Byerly; S R Braddock; M Masuno; K Imaizumi; K Devriendt; M R Eccles
Journal:  Am J Hum Genet       Date:  1997-04       Impact factor: 11.025

6.  CC2D2A is mutated in Joubert syndrome and interacts with the ciliopathy-associated basal body protein CEP290.

Authors:  Nicholas T Gorden; Heleen H Arts; Melissa A Parisi; Karlien L M Coene; Stef J F Letteboer; Sylvia E C van Beersum; Dorus A Mans; Abigail Hikida; Melissa Eckert; Dana Knutzen; Abdulrahman F Alswaid; Hamit Ozyurek; Sel Dibooglu; Edgar A Otto; Yangfan Liu; Erica E Davis; Carolyn M Hutter; Theo K Bammler; Frederico M Farin; Michael Dorschner; Meral Topçu; Elaine H Zackai; Phillip Rosenthal; Kelly N Owens; Nicholas Katsanis; John B Vincent; Friedhelm Hildebrandt; Edwin W Rubel; David W Raible; Nine V A M Knoers; Phillip F Chance; Ronald Roepman; Cecilia B Moens; Ian A Glass; Dan Doherty
Journal:  Am J Hum Genet       Date:  2008-10-23       Impact factor: 11.025

Review 7.  Joubert syndrome: insights into brain development, cilium biology, and complex disease.

Authors:  Dan Doherty
Journal:  Semin Pediatr Neurol       Date:  2009-09       Impact factor: 1.636

8.  MKS3/TMEM67 mutations are a major cause of COACH Syndrome, a Joubert Syndrome related disorder with liver involvement.

Authors:  Francesco Brancati; Miriam Iannicelli; Lorena Travaglini; Annalisa Mazzotta; Enrico Bertini; Eugen Boltshauser; Stefano D'Arrigo; Francesco Emma; Elisa Fazzi; Romina Gallizzi; Mattia Gentile; Damir Loncarevic; Vlatka Mejaski-Bosnjak; Chiara Pantaleoni; Luciana Rigoli; Carmelo D Salpietro; Sabrina Signorini; Gilda Rita Stringini; Alain Verloes; Dominika Zabloka; Bruno Dallapiccola; Joseph G Gleeson; Enza Maria Valente
Journal:  Hum Mutat       Date:  2009-02       Impact factor: 4.878

9.  Mutations in the cilia gene ARL13B lead to the classical form of Joubert syndrome.

Authors:  Vincent Cantagrel; Jennifer L Silhavy; Stephanie L Bielas; Dominika Swistun; Sarah E Marsh; Julien Y Bertrand; Sophie Audollent; Tania Attié-Bitach; Kenton R Holden; William B Dobyns; David Traver; Lihadh Al-Gazali; Bassam R Ali; Tom H Lindner; Tamara Caspary; Edgar A Otto; Friedhelm Hildebrandt; Ian A Glass; Clare V Logan; Colin A Johnson; Christopher Bennett; Francesco Brancati; Enza Maria Valente; C Geoffrey Woods; Joseph G Gleeson
Journal:  Am J Hum Genet       Date:  2008-08       Impact factor: 11.025

10.  Mutations in 3 genes (MKS3, CC2D2A and RPGRIP1L) cause COACH syndrome (Joubert syndrome with congenital hepatic fibrosis).

Authors:  D Doherty; M A Parisi; L S Finn; M Gunay-Aygun; M Al-Mateen; D Bates; C Clericuzio; H Demir; M Dorschner; A J van Essen; W A Gahl; M Gentile; N T Gorden; A Hikida; D Knutzen; H Ozyurek; I Phelps; P Rosenthal; A Verloes; H Weigand; P F Chance; W B Dobyns; I A Glass
Journal:  J Med Genet       Date:  2009-07-01       Impact factor: 6.318

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