AAV2 gene therapy readministration in three adults with congenital blindness. (Q30587265)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22323828%20AND%20SRC:MED&resulttype=core&format=json

22 January 2020

title

AAV2 gene therapy readministration in three adults with congenital blindness (English)

1 reference

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22 January 2020

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8

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22 January 2020

Francesca Simonelli

16

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22 January 2020

Alberto Auricchio

15

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22 January 2020

Katherine A. High

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22 January 2020

20

Albert M Maguire

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22 January 2020

4

Kathleen A Marshall

1 reference

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22 January 2020

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Federico Mingozzi

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22 January 2020

author name string

Jean Bennett

1

1 reference

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22 January 2020

Manzar Ashtari

2

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22 January 2020

Jennifer Wellman

3

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22 January 2020

Laura L Cyckowski

5

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22 January 2020

Daniel C Chung

6

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22 January 2020

Sarah McCague

7

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22 January 2020

Yifeng Chen

9

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22 January 2020

Jeannette L Bennicelli

10

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22 January 2020

Xiaosong Zhu

11

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22 January 2020

Gui-Shuang Ying

12

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22 January 2020

Junwei Sun

13

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22 January 2020

J Fraser Wright

14

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22 January 2020

Kenneth S Shindler

17

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22 January 2020

language of work or name

English

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publication date

1 February 2012

1 reference

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Science Translational Medicine

22 January 2020

published in

1 reference

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22 January 2020

volume

4

1 reference

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22 January 2020

issue

120

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22 January 2020

page(s)

120ra15

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The human visual cortex responds to gene therapy-mediated recovery of retinal function

22 January 2020

cites work

1 reference

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Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies

20 June 2018

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A phase 2 study to evaluate the safety and immunogenicity of a recombinant HIV type 1 vaccine based on adeno-associated virus

20 June 2018

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Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness

20 June 2018

1 reference

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

20 June 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

20 June 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

20 June 2018

1 reference

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Leber congenital amaurosis: genes, proteins and disease mechanisms

20 June 2018

1 reference

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

20 June 2018

1 reference

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

20 June 2018

1 reference

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Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4169122

Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients

20 June 2018

1 reference

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Analysis of functional image analysis contest (FIAC) data with brainvoyager QX: From single-subject to cortically aligned group general linear model analysis and self-organizing group independent component analysis

20 June 2018

1 reference

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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

20 June 2018

1 reference

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Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle.

20 June 2018

1 reference

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Impairment of the transient pupillary light reflex in Rpe65(-/-) mice and humans with leber congenital amaurosis

20 June 2018

1 reference

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Controlling the familywise error rate in functional neuroimaging: a comparative review

20 June 2018

1 reference

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Genetics and phenotypes of RPE65 mutations in inherited retinal degeneration.

20 June 2018

1 reference

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

20 June 2018

1 reference

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

20 June 2018

1 reference

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Mutations in RPE65 cause autosomal recessive childhood–onset severe retinal dystrophy

20 June 2018

1 reference

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

20 June 2018

1 reference

Crossref

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3002865

Gene therapy restores vision in a canine model of childhood blindness

21 January 2018

1 reference

Crossref

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3002865

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

21 January 2018

1 reference

Crossref

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3002865

Early-onset severe rod-cone dystrophy in young children with RPE65 mutations

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22323828

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1126/SCITRANSLMED.3002865

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22323828%20AND%20SRC:MED&resulttype=core&format=json

22 January 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22323828%20AND%20SRC:MED&resulttype=core&format=json

22 January 2020

PubMed publication ID

22323828

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22323828%20AND%20SRC:MED&resulttype=core&format=json