In utero gene therapy rescues vision in a murine model of congenital blindness (Q44753954)

6 December 2017

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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6 December 2017

Enrico M Surace

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author name string

Nadine S Dejneka

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6 December 2017

Tomas S Aleman

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Artur V Cideciyan

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Arkady Lyubarsky

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Andrey Savchenko

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T Michael Redmond

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Waixing Tang

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6 December 2017

Zhangyong Wei

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Tonia S Rex

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Ernest Glover

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Albert M Maguire

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Edward N Pugh

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Jean Bennett

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1 February 2004

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182-188

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Molecular genetics of Leber congenital amaurosis

6 December 2017

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

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

7 January 2021

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

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

7 January 2021

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

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

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

7 January 2021

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

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

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

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

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

7 January 2021

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

In utero gene therapy: transfer and long-term expression of the bacterial neo(r) gene in sheep after direct injection of retroviral vectors into preimmune fetuses

7 January 2021

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

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

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

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

7 January 2021

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

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

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

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

7 January 2021

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

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

7 January 2021

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

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

7 January 2021

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

Retinal rod photoreceptor-specific gene mutation perturbs cone pathway development.

7 January 2021

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

Retinal remodeling triggered by photoreceptor degenerations.

7 January 2021

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

Reorganization of human cortical maps caused by inherited photoreceptor abnormalities.

7 January 2021

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

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

7 January 2021

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

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

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

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

7 January 2021

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

10.1016/J.YMTHE.2003.11.013

7 January 2021

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6 December 2017

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