Literature DB >> 19578016

The natural history of stargardt disease with specific sequence mutation in the ABCA4 gene.

Mohamed A Genead1, Gerald A Fishman, Edwin M Stone, Rando Allikmets.   

Abstract

PURPOSE: To determine longitudinal changes in fundus appearance and visual function in patients with Stargardt with at least one allelic mutation (Gly1961Glu) in the ABCA4 gene.
METHODS: Sixteen patients with a diagnosis of Stargardt disease and a Gly1961Glu mutation were enrolled. All patients underwent a complete ocular examination including best corrected visual acuity, Goldmann visual field (GVF), and full-field ERG examinations. The percentage of patients who showed at least a doubling in the log of the minimum angle of visual resolution (logMAR) between their initial and most recent visits was determined, as was the percentage of patients who showed a doubling in the size of the central scotoma over this duration.
RESULTS: Nine patients had at least a doubling of the logMAR visual acuity in their right eyes and 10 patients in their left eyes, over a mean follow-up (FU) period of 18.6 years. Of 15 patients, 46.7% had equal to or more than a doubling of the central scotoma area in response to a II2e test stimulus in the right eye and 60.0% in the left eyes. Among 10 patients whose ERGs were initially normal for rod and cone responses, 8 remained normal at their most recent FU visit.
CONCLUSIONS: In these patients with Stargardt disease and a Gly1961Glu mutation, most showed a clinical phenotype characterized by fundus changes localized to the foveal and parafoveal regions, normal ERG amplitudes, absence of a silent or masked choroid, and a mean age at initial presentation in the third decade.

Entities:  

Mesh:

Substances:

Year:  2009        PMID: 19578016     DOI: 10.1167/iovs.09-3611

Source DB:  PubMed          Journal:  Invest Ophthalmol Vis Sci        ISSN: 0146-0404            Impact factor:   4.799


  20 in total

1.  Retinal phenotypes in patients homozygous for the G1961E mutation in the ABCA4 gene.

Authors:  Tomas R Burke; Gerald A Fishman; Jana Zernant; Carl Schubert; Stephen H Tsang; R Theodore Smith; Radha Ayyagari; Robert K Koenekoop; Allison Umfress; Maria Laura Ciccarelli; Alfonso Baldi; Alessandro Iannaccone; Frans P M Cremers; Caroline C W Klaver; Rando Allikmets
Journal:  Invest Ophthalmol Vis Sci       Date:  2012-07-03       Impact factor: 4.799

Review 2.  Allelic and phenotypic heterogeneity in ABCA4 mutations.

Authors:  Tomas R Burke; Stephen H Tsang
Journal:  Ophthalmic Genet       Date:  2011-04-21       Impact factor: 1.803

3.  The CFTR Corrector, VX-809 (Lumacaftor), Rescues ABCA4 Trafficking Mutants: a Potential Treatment for Stargardt Disease.

Authors:  Qiangni Liu; Inna Sabirzhanova; Emily Anne Smith Bergbower; Murali Yanda; William G Guggino; Liudmila Cebotaru
Journal:  Cell Physiol Biochem       Date:  2019

4.  Spectral-domain OCT peripapillary retinal nerve fibre layer thickness measurements in patients with Stargardt disease.

Authors:  Mohamed A Genead; Gerald A Fishman; Anastasios Anastasakis
Journal:  Br J Ophthalmol       Date:  2010-10-08       Impact factor: 4.638

Review 5.  Defective lipid transport and biosynthesis in recessive and dominant Stargardt macular degeneration.

Authors:  Robert S Molday; Kang Zhang
Journal:  Prog Lipid Res       Date:  2010-07-13       Impact factor: 16.195

6.  Association of dark-adapted visual function with retinal structural changes in patients with Stargardt disease.

Authors:  Serena Salvatore; Gerald A Fishman; J Jason McAnany; Mohamed A Genead
Journal:  Retina       Date:  2014-05       Impact factor: 4.256

7.  Clinical phenotypes and prognostic full-field electroretinographic findings in Stargardt disease.

Authors:  Sarwar Zahid; Thiran Jayasundera; William Rhoades; Kari Branham; Naheed Khan; Leslie M Niziol; David C Musch; John R Heckenlively
Journal:  Am J Ophthalmol       Date:  2012-12-05       Impact factor: 5.258

8.  Early diagnosis of Stargardt disease with multifocal electroretinogram in children.

Authors:  Anna Praidou; Richard Hagan; William Newman; Arvind Chandna
Journal:  Int Ophthalmol       Date:  2013-06-13       Impact factor: 2.031

9.  CLINICAL CHARACTERIZATION OF STARGARDT DISEASE PATIENTS WITH THE p.N1868I ABCA4 MUTATION.

Authors:  Frederick T Collison; Winston Lee; Gerald A Fishman; Jason C Park; Jana Zernant; J Jason McAnany; Rando Allikmets
Journal:  Retina       Date:  2019-12       Impact factor: 4.256

10.  Deep learning for the segmentation of preserved photoreceptors on en face optical coherence tomography in two inherited retinal diseases.

Authors:  Acner Camino; Zhuo Wang; Jie Wang; Mark E Pennesi; Paul Yang; David Huang; Dengwang Li; Yali Jia
Journal:  Biomed Opt Express       Date:  2018-06-12       Impact factor: 3.732

View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.