Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. (Q37701838)

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scientific article published on 30 June 2016

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English	Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.	scientific article published on 30 June 2016

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scholarly article

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17 March 2020

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial (English)

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17 March 2020

phase I clinical trial

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17 March 2020

Katherine A. High

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17 March 2020

Alberto Auricchio

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Alberto Auricchio

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Francesca Simonelli

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Francesca Simonelli

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Albert M Maguire

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Albert M Maguire

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Kathleen Marshall

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Kathleen A Marshall

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17 March 2020

Federico Mingozzi

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Federico Mingozzi

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author name string

Jean Bennett

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17 March 2020

Jennifer Wellman

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17 March 2020

Sarah McCague

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17 March 2020

Manzar Ashtari

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Julie DiStefano-Pappas

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17 March 2020

Okan U Elci

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17 March 2020

Daniel C Chung

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17 March 2020

Junwei Sun

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17 March 2020

J Fraser Wright

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17 March 2020

Dominique R Cross

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17 March 2020

Puya Aravand

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17 March 2020

Laura L Cyckowski

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17 March 2020

Jeannette L Bennicelli

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17 March 2020

Jason Ruggiero

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17 March 2020

Bart P Leroy

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30 June 2016

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17 March 2020

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17 March 2020

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17 March 2020

661-672

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17 March 2020

Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosis

1 reference

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23 August 2017

Long-term effect of gene therapy on Leber's congenital amaurosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

Improvement and decline in vision with gene therapy in childhood blindness.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

fMRI of retina-originated phosphenes experienced by patients with Leber congenital amaurosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

AAV2 gene therapy readministration in three adults with congenital blindness.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

23 August 2017

Resolving the clinical acuity categories "hand motion" and "counting fingers" using the Freiburg Visual Acuity Test (FrACT).

1 reference

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14 September 2017

Genetics and phenotypes of RPE65 mutations in inherited retinal degeneration.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

28 September 2017

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

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23 June 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

1 September 2018

Gene therapy restores vision in a canine model of childhood blindness

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

1 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5351775

1 September 2018

Visual acuity measurements

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7 January 2021

based on heuristic

inferred from DOI database lookup

Identifiers

10.1016/S0140-6736(16)30371-3

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17 March 2020

1 reference

Europe PubMed Central

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17 March 2020

1 reference

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17 March 2020

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