Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa (Q34158497)

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19 January 2020

title

Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa (English)

1 reference

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autosomal dominant disease

19 January 2020

main subject

gene therapy

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19 January 2020

author name string

Sophia Millington-Ward

1

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19 January 2020

Naomi Chadderton

2

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19 January 2020

Mary O'Reilly

3

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19 January 2020

Arpad Palfi

4

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19 January 2020

Claire Kilty

6

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19 January 2020

Marian Humphries

7

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19 January 2020

Uwe Wolfrum

8

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19 January 2020

Jean Bennett

9

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19 January 2020

Peter Humphries

10

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19 January 2020

Paul F Kenna

11

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19 January 2020

G Jane Farrar

12

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19 January 2020

publication date

11 January 2011

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19 January 2020

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19 January 2020

volume

19

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19 January 2020

issue

4

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19 January 2020

page(s)

642-649

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Prevention of autosomal dominant retinitis pigmentosa by systemic drug therapy targeting heat shock protein 90 (Hsp90)

19 January 2020

cites work

1 reference

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Gene therapeutic approaches for dominant retinopathies

5 July 2018

1 reference

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Viral vector-mediated RNA interference

5 July 2018

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Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78

5 July 2018

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Photoreceptor degeneration: genetic and mechanistic dissection of a complex trait

5 July 2018

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Little vector, big gene transduction: fragmented genome reassembly of adeno-associated virus

5 July 2018

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Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

5 July 2018

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

5 July 2018

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Characterization of genome integrity for oversized recombinant AAV vector

5 July 2018

1 reference

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Effect of genome size on AAV vector packaging

5 July 2018

1 reference

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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

5 July 2018

1 reference

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Ex vivo gene therapy using intravitreal injection of GDNF-secreting mouse embryonic stem cells in a rat model of retinal degeneration.

5 July 2018

1 reference

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Functional cone rescue by RdCVF protein in a dominant model of retinitis pigmentosa

5 July 2018

1 reference

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Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy

5 July 2018

1 reference

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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

5 July 2018

1 reference

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

5 July 2018

1 reference

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Safety and efficacy of gene transfer for Leber's congenital amaurosis

5 July 2018

1 reference

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Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

5 July 2018

1 reference

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RNA interference-mediated suppression and replacement of human rhodopsin in vivo

5 July 2018

1 reference

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

5 July 2018

1 reference

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Mechanisms of cell death in rhodopsin retinitis pigmentosa: implications for therapy

5 July 2018

1 reference

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Electroporation and RNA interference in the rodent retina in vivo and in vitro

5 July 2018

1 reference

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On the genetics of retinitis pigmentosa and on mutation-independent approaches to therapeutic intervention

5 July 2018

1 reference

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

5 July 2018

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Transgenic mice carrying the dominant rhodopsin mutation P347S: evidence for defective vectorial transport of rhodopsin to the outer segments

5 July 2018

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Transgenic mice with a rhodopsin mutation (Pro23His): a mouse model of autosomal dominant retinitis pigmentosa

5 July 2018

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Cytoskeletal elements in arthorpod sensilla and mammalian photoreceptors*

5 July 2018

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In vivo application of an RNAi strategy for the selective suppression of a mutant allele

5 July 2018

1 reference

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Adeno-associated virus-mediated rhodopsin replacement provides therapeutic benefit in mice with a targeted disruption of the rhodopsin gene.

28 October 2018

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Successful RPE65 gene replacement and improved visual function in humans

28 October 2018

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Fast and reliable titration of recombinant adeno-associated virus type-2 using quantitative real-time PCR.

28 October 2018

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Definition of an efficient synthetic poly(A) site

28 October 2018

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12 December 2020

Retinopathy induced in mice by targeted disruption of the rhodopsin gene

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12 December 2020

Strategems in vitro for gene therapies directed to dominant mutations

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12 December 2020

Rhodopsin--advances and perspectives

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12 December 2020

Caspase-1 ablation protects photoreceptors in a model of autosomal dominant retinitis pigmentosa

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12 December 2020

Identifiers

DOI

10.1038/MT.2010.293

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21224835 AND SRC:MED&resulttype=core&format=json

19 January 2020

1 reference

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19 January 2020

PubMed publication ID

21224835

1 reference