Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. (Q28306059)

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5 March 2020

title

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial (English)

1 reference

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5 March 2020

main subject

gene therapy

0 references

phase I clinical trial

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phase II clinical trial

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author

Graeme Black

10

1 reference

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5 March 2020

Robert MacLaren

1

2 references

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5 March 2020

ORCID Public Data File 2021

Len Seymour

6

1 reference

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5 March 2020

Andrew J Lotery

11

1 reference

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5 March 2020

Miguel C. Seabra

14

1 reference

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5 March 2020

9

Frans P M Cremers

1 reference

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5 March 2020

8

Matthew J During

1 reference

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5 March 2020

author name string

Markus Groppe

2

1 reference

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5 March 2020

Alun R Barnard

3

1 reference

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5 March 2020

Charles L Cottriall

4

1 reference

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5 March 2020

Tanya Tolmachova

5

1 reference

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5 March 2020

K Reed Clark

7

1 reference

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5 March 2020

Susan M Downes

12

1 reference

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5 March 2020

Andrew R Webster

13

1 reference

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5 March 2020

language of work or name

English

0 references

publication date

16 January 2014

1 reference

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5 March 2020

1 reference

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volume

383

1 reference

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issue

9923

1 reference

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5 March 2020

page(s)

1129-1137

1 reference

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Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey

5 March 2020

cites work

1 reference

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A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement

20 March 2017

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AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

20 March 2017

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 March 2017

1 reference

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Vision 1 year after gene therapy for Leber's congenital amaurosis

20 March 2017

1 reference

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

20 March 2017

1 reference

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Retinal degeneration in choroideremia: deficiency of rab geranylgeranyl transferase

20 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4171740

Cloning and characterization of the human choroideremia gene

20 March 2017

1 reference

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Cloning of a gene that is rearranged in patients with choroideraemia

20 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4171740

Aberrant splicing of the CHM gene is a significant cause of choroideremia

20 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4171740

Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo

20 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4171740

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4171740

High-resolution images of retinal structure in patients with choroideremia

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4171740

AAV2 gene therapy readministration in three adults with congenital blindness.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4171740

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

29 September 2017

1 reference

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Retinal pigment epithelium defects accelerate photoreceptor degeneration in cell type-specific knockout mouse models of choroideremia

29 September 2017

1 reference

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An analysis of retinal gene therapy clinical trials.

29 September 2017

1 reference

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

29 September 2017

1 reference

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Ciliary neurotrophic factor (CNTF) for human retinal degeneration: phase I trial of CNTF delivered by encapsulated cell intraocular implants

29 September 2017

1 reference

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Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy

29 September 2017

1 reference

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Molecular basis of choroideremia (CHM): mutations involving the Rab escort protein-1 (REP-1) gene

29 September 2017

1 reference

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Test-retest variability of microperimetry using the Nidek MP1 in patients with macular disease

2 June 2018

1 reference

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Remodeling of the Human Retina in Choroideremia: Rab Escort Protein 1 (REP-1) Mutations

27 November 2018

1 reference

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Gene therapy for choroideremia: in vitro rescue mediated by recombinant adenovirus

27 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4171740

Inter-observer variation in refraction and visual acuity measurement using a standardized protocol

27 November 2018

1 reference

Crossref

https://api.crossref.org/works/10.1016/S0140-6736(13)62117-0

inferred from DOI database lookup

7 January 2021

based on heuristic

Case 15-2009: a man with coma after cardiac arrest

1 reference

Crossref

https://api.crossref.org/works/10.1016/S0140-6736(13)62117-0

inferred from DOI database lookup

7 January 2021

based on heuristic

Choroideremia associated with subretinal neovascular membrane

1 reference

Crossref

https://api.crossref.org/works/10.1016/S0140-6736(13)62117-0

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1016/S0140-6736(13)62117-0

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24439297 AND SRC:MED&resulttype=core&format=json

OpenCitations bibliographic resource ID

5 March 2020

584543

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

2 references

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

584543

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24439297 AND SRC:MED&resulttype=core&format=json

5 March 2020

PubMed publication ID

24439297

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24439297 AND SRC:MED&resulttype=core&format=json