Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection (Q45417238)

14 December 2017

Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection (English)

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14 December 2017

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14 December 2017

Gregory M Acland

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14 December 2017

Gustavo D Aguirre

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14 December 2017

Tomas S Aleman

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14 December 2017

Sharon B Schwartz

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14 December 2017

Artur V Cideciyan

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14 December 2017

Caroline J Zeiss

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14 December 2017

Andras M Komaromy

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14 December 2017

Shalesh Kaushal

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14 December 2017

Alejandro J Roman

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14 December 2017

Elizabeth A M Windsor

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14 December 2017

Alexander Sumaroka

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14 December 2017

Susan E Pearce-Kelling

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14 December 2017

Thomas J Conlon

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14 December 2017

Vincent A Chiodo

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14 December 2017

Sanford L Boye

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14 December 2017

Terence R Flotte

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14 December 2017

Albert M Maguire

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14 December 2017

Jean Bennett

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14 December 2017

William W Hauswirth

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14 December 2017

27 April 2006

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14 December 2017

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14 December 2017

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14 December 2017

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14 December 2017

1074-1084

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Molecular genetics of Leber congenital amaurosis

14 December 2017

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Concordance and recessive inheritance of Leber congenital amaurosis

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65-/- knockout mouse eye results in limited rescue

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Recombinant AAV-mediated gene transfer to the retina: gene therapy perspectives

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Recent advances in early-onset severe retinal degeneration: more than just basic research

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Biodistribution of rAAV vectors following intraocular administration: evidence for the presence and persistence of vector DNA in the optic nerve and in the brain.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Postsurgical assessment and long-term safety of recombinant adeno-associated virus-mediated gene transfer into the retinas of dogs and primates

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Distribution of intraocular pressure in dogs

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Surrogate electroretinographic markers for assessing therapeutic efficacy in the retina

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Safety assessment of biotechnology-derived pharmaceuticals: ICH and beyond

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Recent developments in gene transfer: risk and ethics

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Practical considerations of recombinant adeno-associated virus-mediated gene transfer for treatment of retinal degenerations

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Adeno-associated viral vectors for retinal gene transfer.

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Cellular remodeling in mammalian retina: results from studies of experimental retinal detachment

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Basic fibroblast growth factor and local injury protect photoreceptors from light damage in the rat

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV.

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Reporter expression persists 1 year after adeno-associated virus-mediated gene transfer to the optic nerve

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Streamlined large-scale production of recombinant adeno-associated virus (rAAV) vectors

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Naturally occurring rhodopsin mutation in the dog causes retinal dysfunction and degeneration mimicking human dominant retinitis pigmentosa

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Factors affecting the interpretation of canine and nonhuman primate clinical pathology

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Ribozyme rescue of photoreceptor cells in P23H transgenic rats: long-term survival and late-stage therapy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.005

10.1016/J.YMTHE.2006.03.005

7 January 2021

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release_pw2slxgjzna2nfv3t5h7ikjjui

14 December 2017

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24 November 2022

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