Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa. (Q37376742)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23692380%20AND%20SRC:MED&resulttype=core&format=json

1 March 2020

title

Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa (English)

1 reference

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1 March 2020

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2

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1 March 2020

Fowzan S Alkuraya

14

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1 March 2020

William W Hauswirth

16

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1 March 2020

author name string

Thomas J Conlon

1

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1 March 2020

Kirsten Erger

3

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1 March 2020

Travis Cossette

4

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1 March 2020

Ji-jing Pang

5

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1 March 2020

Renee Ryals

6

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1 March 2020

Nathalie Clément

7

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1 March 2020

Brian Cleaver

8

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1 March 2020

Issam McDoom

9

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1 March 2020

Shannon E Boye

10

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1 March 2020

Marc C Peden

11

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1 March 2020

Mark B Sherwood

12

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1 March 2020

Corinne R Abernathy

13

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1 March 2020

Sanford L Boye

15

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1 March 2020

language of work or name

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1 March 2013

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23692380%20AND%20SRC:MED&resulttype=core&format=json

Human gene therapy. Clinical development

1 March 2020

published in

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1 March 2020

volume

24

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1 March 2020

page(s)

23-28

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1 March 2020

issue

1

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A comprehensive review of retinal gene therapy

1 March 2020

cites work

1 reference

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6 September 2017

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A novel MERTK deletion is a common founder mutation in the Faroe Islands and is responsible for a high proportion of retinitis pigmentosa cases

6 September 2017

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Nonsense mutation in MERTK causes autosomal recessive retinitis pigmentosa in a consanguineous Pakistani family

6 September 2017

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Novel mutations in MERTK associated with childhood onset rod-cone dystrophy

6 September 2017

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

6 September 2017

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

6 September 2017

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

6 September 2017

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Adeno-associated viral vectors and the retina

6 September 2017

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6 September 2017

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Clinical characterisation of a family with retinal dystrophy caused by mutation in the Mertk gene

6 September 2017

1 reference

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Inherited retinal dystrophy in the rat.

6 September 2017

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Retinitis pigmentosa and allied diseases: numerous diseases, genes, and inheritance patterns.

6 September 2017

1 reference

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Retinal dystrophy due to paternal isodisomy for chromosome 1 or chromosome 2, with homoallelism for mutations in RPE65 or MERTK, respectively

6 September 2017

1 reference

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Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk

6 September 2017

1 reference

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Mutations in MERTK, the human orthologue of the RCS rat retinal dystrophy gene, cause retinitis pigmentosa

6 September 2017

1 reference

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Mutation of the receptor tyrosine kinase gene Mertk in the retinal dystrophic RCS rat

6 September 2017

1 reference

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Homozygous mutation in MERTK causes severe autosomal recessive retinitis pigmentosa

6 September 2017

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The role of the pigment epithelium in the etiology of inherited retinal dystrophy in the rat

14 September 2017

1 reference

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Focus on molecules: MERTK.

28 September 2017

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26 June 2018

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Characterisation of severe rod-cone dystrophy in a consanguineous family with a splice site mutation in the MERTK gene

2 September 2018

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Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy

2 September 2018

1 reference

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AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa

2 September 2018

1 reference

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Inherited Retinal Dystrophy: Primary Defect in Pigment Epithelium Determined with Experimental Rat Chimeras

2 September 2018

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PubMed

https://pubmed.ncbi.nlm.nih.gov/23692380

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1089/HUMC.2013.037

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23692380%20AND%20SRC:MED&resulttype=core&format=json

1 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23692380%20AND%20SRC:MED&resulttype=core&format=json

1 March 2020

1 reference

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