Literature DB >> 21931134

Histopathology and functional correlations in a patient with a mutation in RPE65, the gene for retinol isomerase.

Vera L Bonilha1, Mary E Rayborn, Yong Li, Gregory H Grossman, Eliot L Berson, Joe G Hollyfield.   

Abstract

PURPOSE: Here the authors describe the structural features of the retina and retinal pigment epithelium (RPE) in postmortem donor eyes of a 56-year-old patient with a homozygous missense RPE65 mutation (Ala132Thr) and correlate the pathology with the patient's visual function last measured at age 51.
METHODS: Eyes were enucleated within 13.5 hours after death. Representative areas from the macula and periphery were processed for light and electron microscopy. Immunofluorescence was used to localize the distribution of RPE65, rhodopsin, and cone arrestin. The autofluorescence in the RPE was compared with that of two normal eyes from age-similar donors.
RESULTS: Histologic examination revealed the loss of rods and cones across most areas of the retina, attenuated retinal vessels, and RPE thinning in both eyes. A small number of highly disorganized cones were present in the macula that showed simultaneous labeling with cone arrestin and red/green or blue opsin. RPE65 immunoreactivity and RPE autofluorescence were reduced compared with control eyes in all areas studied. Rhodopsin labeling was observed in rods in the far periphery. The optic nerve showed a reduced number of axons.
CONCLUSIONS: The clinical findings of reduced visual acuity, constricted fields, and reduced electroretinograms (ERGs) 5 years before death correlated with the small number of cones present in the macula and the extensive loss of photoreceptors in the periphery. The absence of autofluorescence in the RPE suggests that photoreceptor cells were probably missing across the retina for extended periods of time. Possible mechanisms that could lead to photoreceptor cell death are discussed.

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Year:  2011        PMID: 21931134      PMCID: PMC3208160          DOI: 10.1167/iovs.11-7973

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


  60 in total

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Authors:  S K John; J E Smith; G D Aguirre; A H Milam
Journal:  Mol Vis       Date:  2000-11-03       Impact factor: 2.367

2.  Isomerization and oxidation of vitamin a in cone-dominant retinas: a novel pathway for visual-pigment regeneration in daylight.

Authors:  Nathan L Mata; Roxana A Radu; Richard C Clemmons; Gabriel H Travis
Journal:  Neuron       Date:  2002-09-26       Impact factor: 17.173

3.  RPE65 is the isomerohydrolase in the retinoid visual cycle.

Authors:  Gennadiy Moiseyev; Ying Chen; Yusuke Takahashi; Bill X Wu; Jian-Xing Ma
Journal:  Proc Natl Acad Sci U S A       Date:  2005-08-22       Impact factor: 11.205

4.  Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or leber congenital amaurosis.

Authors:  H Morimura; G A Fishman; S A Grover; A B Fulton; E L Berson; T P Dryja
Journal:  Proc Natl Acad Sci U S A       Date:  1998-03-17       Impact factor: 11.205

5.  Peripapillary pigmentary retinal degeneration.

Authors:  K G Noble; R E Carr
Journal:  Am J Ophthalmol       Date:  1978-07       Impact factor: 5.258

6.  Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis.

Authors:  Alejandro J Roman; Sanford L Boye; Tomas S Aleman; Ji-jing Pang; J Hugh McDowell; Shannon E Boye; Artur V Cideciyan; Samuel G Jacobson; William W Hauswirth
Journal:  Mol Vis       Date:  2007-09-18       Impact factor: 2.367

7.  Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.

Authors:  Albert M Maguire; Katherine A High; Alberto Auricchio; J Fraser Wright; Eric A Pierce; Francesco Testa; Federico Mingozzi; Jeannette L Bennicelli; Gui-shuang Ying; Settimio Rossi; Ann Fulton; Kathleen A Marshall; Sandro Banfi; Daniel C Chung; Jessica I W Morgan; Bernd Hauck; Olga Zelenaia; Xiaosong Zhu; Leslie Raffini; Frauke Coppieters; Elfride De Baere; Kenneth S Shindler; Nicholas J Volpe; Enrico M Surace; Carmela Acerra; Arkady Lyubarsky; T Michael Redmond; Edwin Stone; Junwei Sun; Jennifer Wellman McDonnell; Bart P Leroy; Francesca Simonelli; Jean Bennett
Journal:  Lancet       Date:  2009-10-23       Impact factor: 79.321

8.  In utero gene therapy rescues vision in a murine model of congenital blindness.

Authors:  Nadine S Dejneka; Enrico M Surace; Tomas S Aleman; Artur V Cideciyan; Arkady Lyubarsky; Andrey Savchenko; T Michael Redmond; Waixing Tang; Zhangyong Wei; Tonia S Rex; Ernest Glover; Albert M Maguire; Edward N Pugh; Samuel G Jacobson; Jean Bennett
Journal:  Mol Ther       Date:  2004-02       Impact factor: 11.454

9.  Defining the residual vision in leber congenital amaurosis caused by RPE65 mutations.

Authors:  Samuel G Jacobson; Tomas S Aleman; Artur V Cideciyan; Alejandro J Roman; Alexander Sumaroka; Elizabeth A M Windsor; Sharon B Schwartz; Elise Heon; Edwin M Stone
Journal:  Invest Ophthalmol Vis Sci       Date:  2008-12-30       Impact factor: 4.799

10.  Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.

Authors:  Francesca Simonelli; Albert M Maguire; Francesco Testa; Eric A Pierce; Federico Mingozzi; Jeannette L Bennicelli; Settimio Rossi; Kathleen Marshall; Sandro Banfi; Enrico M Surace; Junwei Sun; T Michael Redmond; Xiaosong Zhu; Kenneth S Shindler; Gui-Shuang Ying; Carmela Ziviello; Carmela Acerra; J Fraser Wright; Jennifer Wellman McDonnell; Katherine A High; Jean Bennett; Alberto Auricchio
Journal:  Mol Ther       Date:  2009-12-01       Impact factor: 11.454

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  5 in total

1.  Increased levels of gene therapy may not be beneficial in retinal disease.

Authors:  Ellen Townes-Anderson
Journal:  Proc Natl Acad Sci U S A       Date:  2013-04-03       Impact factor: 11.205

2.  Histopathology of the Retina from a Three Year-Old Suspected to Have Joubert Syndrome.

Authors:  V L Bonilha; M E Rayborn; B A Bell; M J Marino; E I Traboulsi; S A Hagstrom; J G Hollyfield
Journal:  Austin J Clin Ophthalmol       Date:  2015-09-21

Review 3.  Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy.

Authors:  Albert M Maguire; Jean Bennett; Elena M Aleman; Bart P Leroy; Tomas S Aleman
Journal:  Mol Ther       Date:  2020-12-03       Impact factor: 11.454

4.  Mutational screening of LCA genes emphasizing RPE65 in South Indian cohort of patients.

Authors:  Anshuman Verma; Vijayalakshmi Perumalsamy; Shashikant Shetty; Maigi Kulm; Periasamy Sundaresan
Journal:  PLoS One       Date:  2013-09-16       Impact factor: 3.240

5.  Alu complementary DNA is enriched in atrophic macular degeneration and triggers retinal pigmented epithelium toxicity via cytosolic innate immunity.

Authors:  Shinichi Fukuda; Siddharth Narendran; Akhil Varshney; Yosuke Nagasaka; Shao-Bin Wang; Kameshwari Ambati; Ivana Apicella; Felipe Pereira; Benjamin J Fowler; Tetsuhiro Yasuma; Shuichiro Hirahara; Reo Yasuma; Peirong Huang; Praveen Yerramothu; Ryan D Makin; Mo Wang; Kirstie L Baker; Kenneth M Marion; Xiwen Huang; Elmira Baghdasaryan; Meenakshi Ambati; Vidya L Ambati; Daipayan Banerjee; Vera L Bonilha; Genrich V Tolstonog; Ulrike Held; Yuichiro Ogura; Hiroko Terasaki; Tetsuro Oshika; Deepak Bhattarai; Kyung Bo Kim; Sanford H Feldman; J Ignacio Aguirre; David R Hinton; Nagaraj Kerur; Srinivas R Sadda; Gerald G Schumann; Bradley D Gelfand; Jayakrishna Ambati
Journal:  Sci Adv       Date:  2021-09-29       Impact factor: 14.136

  5 in total

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