Safety and efficacy of gene transfer for Leber's congenital amaurosis (Q24634724)

22 June 2017

title

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

1 reference

Jennifer Wellman McDonnell

22 June 2017

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7

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29

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Francesco Testa

Francesco Testa

9

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Enrico M. Surace

Enrico M Surace

10

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Francesca Simonelli

Francesca Simonelli

2

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Eric A Pierce

Eric A Pierce

3

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Alberto Auricchio

Alberto Auricchio

30

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Sandro Banfi

Sandro Banfi

7

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Settimio Rossi

11

Settimio Rossi

1 reference

22 June 2017

1

Albert M Maguire

1 reference

22 June 2017

8

Kathleen A Marshall

1 reference

22 June 2017

5

Federico Mingozzi

1 reference

22 June 2017

Edward N Pugh

4

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22 June 2017

Jeannette Bennicelli

6

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22 June 2017

Arkady Lyubarsky

12

1 reference

22 June 2017

Valder R Arruda

13

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22 June 2017

Barbara Konkle

14

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22 June 2017

Edwin Stone

15

1 reference

22 June 2017

Junwei Sun

16

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22 June 2017

Jonathan Jacobs

17

1 reference

22 June 2017

Lou Dell'Osso

18

1 reference

22 June 2017

Richard Hertle

19

1 reference

22 June 2017

Jian-xing Ma

20

1 reference

22 June 2017

T Michael Redmond

21

1 reference

22 June 2017

Xiaosong Zhu

22

1 reference

22 June 2017

Bernd Hauck

23

1 reference

22 June 2017

Olga Zelenaia

24

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22 June 2017

Kenneth S Shindler

25

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22 June 2017

Maureen G Maguire

26

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22 June 2017

J Fraser Wright

27

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22 June 2017

Nicholas J Volpe

28

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22 June 2017

Katherine A High

31

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22 June 2017

Jean Bennett

32

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22 June 2017

Jennifer Wellman McDonnell

29

1 reference

22 June 2017

language of work or name

English

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publication date

27 April 2008

1 reference

The New England Journal of Medicine

22 June 2017

published in

1 reference

22 June 2017

volume

358

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22 June 2017

issue

21

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22 June 2017

page(s)

2240-2248

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RPE65 is the isomerohydrolase in the retinoid visual cycle

22 June 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

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

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

Rpe65 is the retinoid isomerase in bovine retinal pigment epithelium

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

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

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

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

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

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

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

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

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

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

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

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

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

Leber congenital amaurosis

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

Congenital stationary night blindness in the dog: common mutation in the RPE65 gene indicates founder effect.

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

The Briard dog: a new animal model of congenital stationary night blindness

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

Daily variation and effects of ambient light and circadian factors on the human light reflex

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

Visual acuity as an outcome measure in clinical trials of retinal diseases

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: implications for the ocular motor system

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

Gene therapy restores vision in a canine model of childhood blindness

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2829748

Test-retest reliability of the multifocal electroretinogram and humphrey visual fields in patients with retinitis pigmentosa

28 November 2018

1 reference

12 December 2020

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

1 reference

12 December 2020

Latency of the pupil light reflex: sample rate, stimulus intensity, and variation in normal subjects

1 reference

12 December 2020

Natural course of retinitis pigmentosa over a three-year interval

1 reference

12 December 2020

Pupillometric measures of retinal sensitivity in infants and adults with retinitis pigmentosa

1 reference

12 December 2020

Idiopathic senile macular hole. Its early stages and pathogenesis

1 reference

12 December 2020

Identifiers

DOI

10.1056/NEJMOA0802315

1 reference

OpenCitations bibliographic resource ID

22 June 2017

163515

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

163515

1 reference