(Q36951825)

17 August 2017

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title

Rescue of sight by gene therapy - closer than it may appear (English)

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17 August 2017

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Tonia S Rex

1

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17 August 2017

1 September 2007

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17 August 2017

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17 August 2017

28

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17 August 2017

3

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17 August 2017

127-133

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A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus

17 August 2017

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

bcl-2 overexpression reduces apoptotic photoreceptor cell death in three different retinal degenerations

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Immune response following intraocular delivery of recombinant viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Sporadic bilateral retinoblastoma and 13q- chromosomal deletion

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Metastatic and nonmetastatic models of retinoblastoma

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Suicide gene therapy for treatment of retinoblastoma in a murine model

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Response of retinoblastoma with vitreous tumor seeding to adenovirus-mediated delivery of thymidine kinase followed by ganciclovir

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Molecular genetics of AMD and current animal models

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Adenovirus-mediated delivery of catalase to retinal pigment epithelial cells protects neighboring photoreceptors from photo-oxidative stress

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Intraocular gene transfer of pigment epithelium-derived factor rescues photoreceptors from light-induced cell death

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

AAV-mediated expression of vascular endothelial growth factor induces choroidal neovascularization in rat.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Overexpression of vascular endothelial growth factor (VEGF) in the retinal pigment epithelium leads to the development of choroidal neovascularization

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Increased expression of VEGF in retinal pigmented epithelial cells is not sufficient to cause choroidal neovascularization.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Periocular injection of an adenoviral vector encoding pigment epithelium-derived factor inhibits choroidal neovascularization

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Pigment epithelium-derived factor inhibits retinal and choroidal neovascularization

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Adenoviral vector-delivered pigment epithelium-derived factor for neovascular age-related macular degeneration: results of a phase I clinical trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Rpe65 is the retinoid isomerase in bovine retinal pigment epithelium

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

RPE65 is the isomerohydrolase in the retinoid visual cycle

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

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

https://api.crossref.org/works/10.1080%2F13816810701503707

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

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

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

In vivo gene therapy in young and adult RPE65-/- dogs produces long-term visual improvement.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Long-term evaluation of retinal function in Prph2Rd2/Rd2 mice following AAV-mediated gene replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Gene replacement therapy in the retinal degeneration slow (rds) mouse: the effect on retinal degeneration following partial transduction of the retina

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

In contrast to AAV-mediated Cntf expression, AAV-mediated Gdnf expression enhances gene replacement therapy in rodent models of retinal degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Encapsidated adenovirus mini-chromosome-mediated delivery of genes to the retina: application to the rescue of photoreceptor degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Long-term protection of retinal structure but not function using RAAV.CNTF in animal models of retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

From vivarium to bedside: lessons learned from animal models.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

New recombinant serotypes of AAV vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Toward a gene therapy for dominant disease: validation of an RNA interference-based mutation-independent approach

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Towards mutation-independent silencing of genes involved in retinal degeneration by RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

RNAi-based suppression and replacement of rds-peripherin in retinal organotypic culture

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Regulatable gene expression systems for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F13816810701503707

10.1080/13816810701503707

21 January 2018

Identifiers

DOI

1 reference

17 August 2017

1 reference

PubMed ID

17896310