RPE65 gene therapy slows cone loss in Rpe65-deficient dogs (Q45881587)

17 December 2017

title

RPE65 gene therapy slows cone loss in Rpe65-deficient dogs (English)

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17 December 2017

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7

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author name string

F M Mowat

1

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17 December 2017

A R Breuwer

2

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J T Bartoe

3

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M J Annear

4

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Z Zhang

5

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A J Smith

6

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S M Petersen-Jones

8

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R R Ali

9

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publication date

6 September 2012

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17 December 2017

published in

Gene Therapy

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17 December 2017

volume

20

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issue

5

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17 December 2017

page(s)

545-555

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17 December 2017

Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle.

cites work

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Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle.

21 January 2018

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RPE65, visual cycle retinol isomerase, is not inherently 11-cis-specific: support for a carbocation mechanism of retinol isomerization

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RPE65 is the isomerohydrolase in the retinoid visual cycle

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Rpe65 is the retinoid isomerase in bovine retinal pigment epithelium

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Intra-retinal visual cycle required for rapid and complete cone dark adaptation.

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An alternative pathway mediates the mouse and human cone visual cycle.

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The cone-specific visual cycle

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New views on RPE65 deficiency: the rod system is the source of vision in a mouse model of Leber congenital amaurosis.

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Cone opsin mislocalization in Rpe65-/- mice: a defect that can be corrected by 11-cis retinal

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Nrl-knockout mice deficient in Rpe65 fail to synthesize 11-cis retinal and cone outer segments

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Regeneration of Photopigment Is Enhanced in Mouse Cone Photoreceptors Expressing RPE65 Protein

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Longitudinal and cross-sectional study of patients with early-onset severe retinal dystrophy associated with RPE65 mutations

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Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation

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Human cone photoreceptor dependence on RPE65 isomerase

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

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Photoreceptor layer topography in children with leber congenital amaurosis caused by RPE65 mutations

21 January 2018

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Retinal disease in Rpe65-deficient mice: comparison to human leber congenital amaurosis due to RPE65 mutations

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Rapid restoration of visual pigment and function with oral retinoid in a mouse model of childhood blindness

21 January 2018

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In conditions of limited chromophore supply rods entrap 11-cis-retinal leading to loss of cone function and cell death

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Mutations in RPE65 cause autosomal recessive childhood–onset severe retinal dystrophy

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Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success

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Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients

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A comprehensive clinical and biochemical functional study of a novel RPE65 hypomorphic mutation

21 January 2018

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Loss of cone photoreceptors caused by chromophore depletion is partially prevented by the artificial chromophore pro-drug, 9-cis-retinyl acetate

21 January 2018

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Differential macular morphology in patients with RPE65-, CEP290-, GUCY2D-, and AIPL1-related Leber congenital amaurosis

21 January 2018

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Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

21 January 2018

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Effect of gene therapy on visual function in Leber's congenital amaurosis

21 January 2018

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Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

21 January 2018

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Vision 1 year after gene therapy for Leber's congenital amaurosis

21 January 2018

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Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial

21 January 2018

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

21 January 2018

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Safety and efficacy of gene transfer for Leber's congenital amaurosis

21 January 2018

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

21 January 2018

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Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis

21 January 2018

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Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.

21 January 2018

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Gene therapy regenerates protein expression in cone photoreceptors in Rpe65(R91W/R91W) mice

21 January 2018

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The Briard dog: a new animal model of congenital stationary night blindness

21 January 2018

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Retinal dystrophy of Swedish briard/briard-beagle dogs is due to a 4-bp deletion in RPE65.

21 January 2018

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Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness

21 January 2018

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Ultrastructural changes of the retina and the retinal pigment epithelium in Briard dogs with hereditary congenital night blindness and partial day blindness

21 January 2018

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Altered expression of retinal molecular markers in the canine RPE65 model of Leber congenital amaurosis

21 January 2018

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Improvement of visual performance with intravitreal administration of 9-cis-retinal in Rpe65-mutant dogs.

21 January 2018

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Gene therapy in the second eye of RPE65-deficient dogs improves retinal function

21 January 2018

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Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium

21 January 2018

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Trafficking of membrane-associated proteins to cone photoreceptor outer segments requires the chromophore 11-cis-retinal

21 January 2018

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Rpe65-/- and Lrat-/- mice: comparable models of leber congenital amaurosis

21 January 2018

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Distribution and morphology of human cone photoreceptors stained with anti-blue opsin

21 January 2018

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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years

21 January 2018

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Downregulation of cone-specific gene expression and degeneration of cone photoreceptors in the Rpe65-/- mouse at early ages

21 January 2018

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Spatial and temporal differences between the expression of short- and middle-wave sensitive cone pigments in the mouse retina: a developmental study

21 January 2018

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The patterning and onset of opsin expression in vertebrate retinae

21 January 2018

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Distribution and development of short-wavelength cones differ between Macaca monkey and human fovea

21 January 2018

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Cone outer segment morphology and cone function in the Rpe65-/- Nrl-/- mouse retina are amenable to retinoid replacement.

21 January 2018

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Cone opsin determines the time course of cone photoreceptor degeneration in Leber congenital amaurosis

21 January 2018

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The mammalian cone visual cycle promotes rapid M/L-cone pigment regeneration independently of the interphotoreceptor retinoid-binding protein

21 January 2018

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R91W mutation in Rpe65 leads to milder early-onset retinal dystrophy due to the generation of low levels of 11-cis-retinal

21 January 2018

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Identification and characterization of rod-derived cone viability factor

21 January 2018

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High-titer adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus

21 January 2018

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Gene expression networks underlying retinoic acid-induced differentiation of human retinoblastoma cells

21 January 2018

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

21 January 2018

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7 January 2021

Case 15-2009: a man with coma after cardiac arrest

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7 January 2021

Retinal dystrophy or 'congenital stationary night blindness' in the Briard dog

1 reference

7 January 2021

Slowly progressive changes of the retina and retinal pigment epithelium in Briard dogs with hereditary retinal dystrophy. A morphological study

1 reference

7 January 2021

Identifiers

DOI

10.1038/GT.2012.63

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17 December 2017

Dimensions Publication ID

1013445754

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17 December 2017