Literature DB >> 16814838

Cortical visual function in the rd12 mouse model of Leber Congenital Amarousis (LCA) after gene replacement therapy to restore retinal function.

S Nusinowitz1, W H Ridder, J J Pang, B Chang, S M Noorwez, S Kaushal, W W Hauswirth, J R Heckenlively.   

Abstract

One eye of rd12 mice received a sub-retinal injection of a vector carrying normal human RPE65 cDNA at post-natal day 18, and at 6- and 13-months of age. Electroretinograms (ERGs) and visual-evoked potentials (VEPs) were recorded to luminance, and to spatially and temporally modulated stimuli to assess the consequences of delayed treatment on visual pathway function. Early treatment resulted in better overall retinal rescue and better rescue of cone-mediated function. VEPs to low temporal frequency luminance modulation were well preserved at all but the oldest treatment age and corresponded to predictions based on the amount of retinal rescue. In contrast, VEPs to high frequency spatially and temporally modulated stimuli were impaired even at the earliest age. These results provide further support that early treatment in human LCA will have the most hope for optimal visual performance.

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Year:  2006        PMID: 16814838     DOI: 10.1016/j.visres.2006.05.010

Source DB:  PubMed          Journal:  Vision Res        ISSN: 0042-6989            Impact factor:   1.886


  20 in total

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

Authors:  Vera L Bonilha; Mary E Rayborn; Yong Li; Gregory H Grossman; Eliot L Berson; Joe G Hollyfield
Journal:  Invest Ophthalmol Vis Sci       Date:  2011-10-28       Impact factor: 4.799

2.  Subretinal delivery and electroporation in pigmented and nonpigmented adult mouse eyes.

Authors:  John M Nickerson; Penny Goodman; Micah A Chrenek; Christiana J Bernal; Lennart Berglin; T Michael Redmond; Jeffrey H Boatright
Journal:  Methods Mol Biol       Date:  2012

Review 3.  Gene therapy for ocular diseases.

Authors:  Melissa M Liu; Jingsheng Tuo; Chi-Chao Chan
Journal:  Br J Ophthalmol       Date:  2010-08-23       Impact factor: 4.638

Review 4.  AAV-mediated gene therapy in mouse models of recessive retinal degeneration.

Authors:  J-J Pang; L Lei; X Dai; W Shi; X Liu; A Dinculescu; J H McDowell
Journal:  Curr Mol Med       Date:  2012-03       Impact factor: 2.222

Review 5.  RPE65: role in the visual cycle, human retinal disease, and gene therapy.

Authors:  Xue Cai; Shannon M Conley; Muna I Naash
Journal:  Ophthalmic Genet       Date:  2009-06       Impact factor: 1.803

6.  An Alternative and Validated Injection Method for Accessing the Subretinal Space via a Transcleral Posterior Approach.

Authors:  Sachin Parikh; Andrew Le; Julian Davenport; Michael B Gorin; Steven Nusinowitz; Anna Matynia
Journal:  J Vis Exp       Date:  2016-12-07       Impact factor: 1.355

7.  Republished review: Gene therapy for ocular diseases.

Authors:  Melissa M Liu; Jingsheng Tuo; Chi-Chao Chan
Journal:  Postgrad Med J       Date:  2011-07       Impact factor: 2.401

Review 8.  Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy.

Authors:  Artur V Cideciyan
Journal:  Prog Retin Eye Res       Date:  2010-04-24       Impact factor: 21.198

9.  Biocompatibility of a Synthetic Biopolymer for the Treatment of Rhegmatogenous Retinal Detachment.

Authors:  Shanta Sarfare; Yann Dacquay; Syed Askari; Steven Nusinowitz; Jean-Pierre Hubschman
Journal:  J Clin Exp Ophthalmol       Date:  2015-09-14

10.  Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement.

Authors:  Artur V Cideciyan; Samuel G Jacobson; William A Beltran; Alexander Sumaroka; Malgorzata Swider; Simone Iwabe; Alejandro J Roman; Melani B Olivares; Sharon B Schwartz; András M Komáromy; William W Hauswirth; Gustavo D Aguirre
Journal:  Proc Natl Acad Sci U S A       Date:  2013-01-22       Impact factor: 11.205

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