Gene therapy restores vision and delays degeneration in the CNGB1(-/-) mouse model of retinitis pigmentosa. (Q45880504)

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8 February 2020

title

Gene therapy restores vision and delays degeneration in the CNGB1(-/-) mouse model of retinitis pigmentosa (English)

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8 February 2020

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5

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8 February 2020

12

Stylianos Michalakis

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8 February 2020

10

Martin Biel

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8 February 2020

author name string

Susanne Koch

1

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8 February 2020

Vithiyanjali Sothilingam

2

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8 February 2020

Marina Garcia Garrido

3

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8 February 2020

Naoyuki Tanimoto

4

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8 February 2020

Fred Koch

6

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8 February 2020

Christina Seide

7

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8 February 2020

Susanne C Beck

8

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8 February 2020

Mathias W Seeliger

9

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8 February 2020

Regine Mühlfriedel

11

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8 February 2020

publication date

16 July 2012

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8 February 2020

published in

Human Molecular Genetics

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volume

21

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8 February 2020

issue

20

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8 February 2020

page(s)

4486-4496

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8 February 2020

1 reference

Retinitis pigmentosa and other dystrophies.

21 January 2018

1 reference

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

21 January 2018

1 reference

Function and dysfunction of CNG channels: insights from channelopathies and mouse models

21 January 2018

1 reference

Cyclic nucleotide-gated channels

21 January 2018

1 reference

Impaired channel targeting and retinal degeneration in mice lacking the cyclic nucleotide-gated channel subunit CNGB1

21 January 2018

1 reference

Knockout of GARPs and the β-subunit of the rod cGMP-gated channel disrupts disk morphogenesis and rod outer segment structural integrity.

21 January 2018

1 reference

Effect of genome size on AAV vector packaging

21 January 2018

1 reference

Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.

21 January 2018

1 reference

Characterization of genome integrity for oversized recombinant AAV vector

21 January 2018

1 reference

Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus

21 January 2018

1 reference

High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors

21 January 2018

1 reference

In vivo confocal imaging of the retina in animal models using scanning laser ophthalmoscopy

21 January 2018

1 reference

Noninvasive, in vivo assessment of mouse retinal structure using optical coherence tomography

21 January 2018

1 reference

Anatomical correlates to the bands seen in the outer retina by optical coherence tomography: literature review and model

21 January 2018

1 reference

Impaired opsin targeting and cone photoreceptor migration in the retina of mice lacking the cyclic nucleotide-gated channel CNGA3.

21 January 2018

1 reference

Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies

21 January 2018

1 reference

Gene supplementation therapy for recessive forms of inherited retinal dystrophies.

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

1 reference

Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy

21 January 2018

1 reference

Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration

21 January 2018

1 reference

AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation

21 January 2018

1 reference

Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.

21 January 2018

1 reference

How vision begins: an odyssey

21 January 2018

1 reference

Restoration of cone vision in the CNGA3-/- mouse model of congenital complete lack of cone photoreceptor function

21 January 2018

1 reference

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

21 January 2018

1 reference

Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column.

21 January 2018

1 reference

Production and characterization of adeno-associated viral vectors

21 January 2018

1 reference

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

21 January 2018

1 reference

Vision tests in the mouse: Functional phenotyping with electroretinography

21 January 2018

1 reference

Photoreceptor peripherin is the normal product of the gene responsible for retinal degeneration in the rds mouse

21 January 2018

1 reference

Chemical chaperone TUDCA preserves cone photoreceptors in a mouse model of Leber congenital amaurosis

21 January 2018

1 reference

The cGMP-gated channel and related glutamic acid-rich proteins interact with peripherin-2 at the rim region of rod photoreceptor disc membranes

21 January 2018

1 reference

Developments of a water-maze procedure for studying spatial learning in the rat

21 January 2018

1 reference

Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.1093/HMG/DDS290

1 reference

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release_e5k5lxs6wzbcdgo26dtvepqmqi

8 February 2020

Fatcat ID

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Fatcat

https://api.fatcat.wiki/v0/release/e5k5lxs6wzbcdgo26dtvepqmqi

mapped directly with Wikidata item

24 November 2022

based on heuristic

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