Literature DB >> 21366429

Macular dystrophy in Heimler syndrome.

Luiz H Lima1, Irene A Barbazetto, Royce Chen, Lawrence A Yannuzzi, Stephen H Tsang, Richard F Spaide.   

Abstract

PURPOSE: To describe the retinal imaging findings in the index patient with Heimler syndrome (OMIM #234580).
DESIGN: Non-interventional case report.
METHODS: A 29-year-old woman with Heimler syndrome developed bilateral vision loss. Fluorescein angiography (FA), fundus autofluorescence (FAF), spectral domain optical coherence tomography (SD-OCT) and electroretinography (ERG) were performed to assess the retinal anatomy and function.
RESULTS: FA showed mottling of the retinal pigment epithelium (RPE) in the posterior pole and periphery of the retina. FAF revealed hyper and hypoautofluorescent dots corresponding to the RPE mottling observed on FA. SD-OCT documented loss of the inner/outer segments boundary, and RPE thinning. ERG testing excluded generalized rod-cone dysfunction.
CONCLUSION: We report an adult-onset macular dystrophy in one of the previously reported patients with Heimler syndrome and hypothesize that this syndrome is probably an expression of a ciliopathy.

Entities:  

Mesh:

Year:  2011        PMID: 21366429      PMCID: PMC3093430          DOI: 10.3109/13816810.2010.551797

Source DB:  PubMed          Journal:  Ophthalmic Genet        ISSN: 1381-6810            Impact factor:   1.803


  16 in total

1.  Amelogenesis imperfecta, sensorineural hearing loss, and Beau's lines, a second case report of Heimler's syndrome.

Authors:  M Tischkowitz; C Clenaghan; S Davies; L Hunter; J Potts; S Verhoef
Journal:  J Med Genet       Date:  1999-12       Impact factor: 6.318

2.  Sensorineural hearing loss and enamel hypoplasia with subtle nail findings: another family with Heimler's syndrome.

Authors:  C Pollak; M Floy; B Say
Journal:  Clin Dysmorphol       Date:  2003-01       Impact factor: 0.816

Review 3.  Development and disease of the photoreceptor cilium.

Authors:  V Ramamurthy; M Cayouette
Journal:  Clin Genet       Date:  2009-08       Impact factor: 4.438

4.  Sensorineural hearing loss, enamel hypoplasia, and nail abnormalities in sibs.

Authors:  A Heimler; J E Fox; J E Hershey; P Crespi
Journal:  Am J Med Genet       Date:  1991-05-01

5.  Primary cilia regulate Shh activity in the control of molar tooth number.

Authors:  Atsushi Ohazama; Courtney J Haycraft; Maisa Seppala; James Blackburn; Sarah Ghafoor; Martyn Cobourne; David C Martinelli; Chen-Ming Fan; Renata Peterkova; Herve Lesot; Bradley K Yoder; Paul T Sharpe
Journal:  Development       Date:  2009-02-11       Impact factor: 6.868

Review 6.  Ellis-van Creveld syndrome and Weyers acrodental dysostosis are caused by cilia-mediated diminished response to hedgehog ligands.

Authors:  Victor L Ruiz-Perez; Judith A Goodship
Journal:  Am J Med Genet C Semin Med Genet       Date:  2009-11-15       Impact factor: 3.908

Review 7.  Making sense of cilia in disease: the human ciliopathies.

Authors:  Kate Baker; Philip L Beales
Journal:  Am J Med Genet C Semin Med Genet       Date:  2009-11-15       Impact factor: 3.908

8.  Age-related cone abnormalities in zebrafish with genetic lesions in sonic hedgehog.

Authors:  Deborah L Stenkamp; Rosanna Satterfield; Kalyani Muhunthan; Tshering Sherpa; Thomas S Vihtelic; David A Cameron
Journal:  Invest Ophthalmol Vis Sci       Date:  2008-05-23       Impact factor: 4.799

9.  Hedgehog signaling regulates sensory cell formation and auditory function in mice and humans.

Authors:  Elizabeth Carroll Driver; Shannon P Pryor; Patrick Hill; Joyce Turner; Ulrich Rüther; Leslie G Biesecker; Andrew J Griffith; Matthew W Kelley
Journal:  J Neurosci       Date:  2008-07-16       Impact factor: 6.167

10.  The dynamic cilium in human diseases.

Authors:  Anna D'Angelo; Brunella Franco
Journal:  Pathogenetics       Date:  2009-05-13
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  6 in total

Review 1.  Characterization of Severity in Zellweger Spectrum Disorder by Clinical Findings: A Scoping Review, Meta-Analysis and Medical Chart Review.

Authors:  Mousumi Bose; Christine Yergeau; Yasmin D'Souza; David D Cuthbertson; Melisa J Lopez; Alyssa K Smolen; Nancy E Braverman
Journal:  Cells       Date:  2022-06-10       Impact factor: 7.666

2.  Spectrum of PEX1 and PEX6 variants in Heimler syndrome.

Authors:  Claire E L Smith; James A Poulter; Alex V Levin; Jenina E Capasso; Susan Price; Tamar Ben-Yosef; Reuven Sharony; William G Newman; Roger C Shore; Steven J Brookes; Alan J Mighell; Chris F Inglehearn
Journal:  Eur J Hum Genet       Date:  2016-06-15       Impact factor: 4.246

3.  Mild form of Zellweger Spectrum Disorders (ZSD) due to variants in PEX1: Detailed clinical investigation in a 9-years-old female.

Authors:  Maria Rosaria Barillari; Marianthi Karali; Valentina Di Iorio; Maria Contaldo; Vincenzo Piccolo; Maria Esposito; Giuseppe Costa; Giuseppe Argenziano; Rosario Serpico; Marco Carotenuto; Gerarda Cappuccio; Sandro Banfi; Paolo Melillo; Francesca Simonelli
Journal:  Mol Genet Metab Rep       Date:  2020-06-20

Review 4.  Dysfunctional peroxisomal lipid metabolisms and their ocular manifestations.

Authors:  Chuck T Chen; Zhuo Shao; Zhongjie Fu
Journal:  Front Cell Dev Biol       Date:  2022-09-07

5.  The peroxisomal disorder spectrum and Heimler syndrome: Deep phenotyping and review of the literature.

Authors:  Malena Daich Varela; Priyam Jani; Wadih M Zein; Precilla D'Souza; Lynne Wolfe; Jennifer Chisholm; Christopher Zalewski; David Adams; Blake M Warner; Laryssa A Huryn; Robert B Hufnagel
Journal:  Am J Med Genet C Semin Med Genet       Date:  2020-08-31       Impact factor: 3.359

6.  Heimler Syndrome Is Caused by Hypomorphic Mutations in the Peroxisome-Biogenesis Genes PEX1 and PEX6.

Authors:  Ilham Ratbi; Kim D Falkenberg; Manou Sommen; Nada Al-Sheqaih; Soukaina Guaoua; Geert Vandeweyer; Jill E Urquhart; Kate E Chandler; Simon G Williams; Neil A Roberts; Mustapha El Alloussi; Graeme C Black; Sacha Ferdinandusse; Hind Ramdi; Audrey Heimler; Alan Fryer; Sally-Ann Lynch; Nicola Cooper; Kai Ren Ong; Claire E L Smith; Christopher F Inglehearn; Alan J Mighell; Claire Elcock; James A Poulter; Marc Tischkowitz; Sally J Davies; Abdelaziz Sefiani; Aleksandr A Mironov; William G Newman; Hans R Waterham; Guy Van Camp
Journal:  Am J Hum Genet       Date:  2015-09-17       Impact factor: 11.025

  6 in total

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