(Q44753954)

6 December 2017

title

In utero gene therapy rescues vision in a murine model of congenital blindness (English)

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6 December 2017

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14

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6 December 2017

Enrico M Surace

2

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author name string

Nadine S Dejneka

1

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6 December 2017

Tomas S Aleman

3

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6 December 2017

Artur V Cideciyan

4

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6 December 2017

Arkady Lyubarsky

5

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Andrey Savchenko

6

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6 December 2017

T Michael Redmond

7

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6 December 2017

Waixing Tang

8

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6 December 2017

Zhangyong Wei

9

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6 December 2017

Tonia S Rex

10

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6 December 2017

Ernest Glover

11

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6 December 2017

Albert M Maguire

12

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6 December 2017

Edward N Pugh

13

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6 December 2017

Jean Bennett

15

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6 December 2017

publication date

1 February 2004

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6 December 2017

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6 December 2017

volume

9

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6 December 2017

issue

2

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6 December 2017

page(s)

182-188

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Molecular genetics of Leber congenital amaurosis

6 December 2017

cites work

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2003.11.013

Mutations in RPE65 cause Leber's congenital amaurosis

7 January 2021

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

7 January 2021

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

7 January 2021

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Rat messenger RNA for the retinal pigment epithelium-specific protein RPE65 gradually accumulates in two weeks from late embryonic days

7 January 2021

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Correlation of regenerable opsin with rod ERG signal in Rpe65-/- mice during development and aging

7 January 2021

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Prenatal human ocular degeneration occurs in Leber's congenital amaurosis (LCA2).

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2003.11.013

Crumbs homolog 1 (CRB1) mutations result in a thick human retina with abnormal lamination

7 January 2021

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Sustained delivery of therapeutic concentrations of human clotting factor IX--a comparison of adenoviral and AAV vectors administered in utero

7 January 2021

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Delivery of adeno-associated virus vectors to the fetal retina: impact of viral capsid proteins on retinal neuronal progenitor transduction

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2003.11.013

In utero gene transfer into the pulmonary epithelium

7 January 2021

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Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches

7 January 2021

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In UteroGene Therapy: Transfer and Long-Term Expression of the BacterialneorGene in Sheep after Direct Injection of Retroviral Vectors into Preimmune Fetuses

7 January 2021

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Ultrasound-guided percutaneous delivery of adenoviral vectors encoding the beta-galactosidase and human factor IX genes to early gestation fetal sheep in utero

7 January 2021

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In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor

7 January 2021

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Adenoviral expression of protein-L-isoaspartyl methyltransferase (PIMT) partially attenuates the biochemical changes in PIMT-deficient mice

7 January 2021

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Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero

7 January 2021

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In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression

7 January 2021

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Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.

7 January 2021

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

7 January 2021

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Infantile and childhood retinal blindness: a molecular perspective (The Franceschetti Lecture)

7 January 2021

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Retinal rod photoreceptor-specific gene mutation perturbs cone pathway development

7 January 2021

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Retinal remodeling triggered by photoreceptor degenerations.

7 January 2021

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Reorganization of human cortical maps caused by inherited photoreceptor abnormalities.

7 January 2021

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Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer

7 January 2021

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Augmented rod bipolar cell function in partial receptor loss: an ERG study in P23H rhodopsin transgenic and aging normal rats

7 January 2021

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An alternative phototransduction model for human rod and cone ERG a-waves: normal parameters and variation with age.

7 January 2021

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Isorhodopsin II: artificial photosensitive pigment formed from 9,13-dicis retinal

7 January 2021

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10.1016/J.YMTHE.2003.11.013

7 January 2021

Identifiers

DOI

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6 December 2017

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