Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia. (Q36852220)

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scientific article published on March 2016

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English	Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.	scientific article published on March 2016

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scholarly article

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27003752 AND SRC:MED&resulttype=core&format=json

30 March 2020

Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia (English)

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Europe PubMed Central

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30 March 2020

William W Hauswirth

14

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30 March 2020

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Guo-jie Ye

1

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30 March 2020

Ewa Budzynski

2

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30 March 2020

Peter Sonnentag

3

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30 March 2020

T Michael Nork

4

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30 March 2020

Paul E Miller

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30 March 2020

Leslie McPherson

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30 March 2020

James N Ver Hoeve

7

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30 March 2020

Leia M Smith

8

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30 March 2020

Tara Arndt

9

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30 March 2020

Savitri Mandapati

10

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30 March 2020

Paulette M Robinson

11

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30 March 2020

Roberto Calcedo

12

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30 March 2020

David R Knop

13

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30 March 2020

Jeffrey D Chulay

15

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30 March 2020

publication date

1 March 2016

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30 March 2020

Human gene therapy. Clinical development

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Europe PubMed Central

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30 March 2020

27

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30 March 2020

1

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30 March 2020

27-36

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30 March 2020

Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

Safety and Biodistribution Evaluation of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin, in RS1-Deficient Mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

Mutations in the unfolded protein response regulator ATF6 cause the cone dysfunction disorder achromatopsia

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

A nonsense mutation in PDE6H causes autosomal-recessive incomplete achromatopsia

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

Gene therapy rescues cone function in congenital achromatopsia

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

A homologous genetic basis of the murine cpfl1 mutant and human achromatopsia linked to mutations in the PDE6C gene

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

Impaired cone function and cone degeneration resulting from CNGB3 deficiency: down-regulation of CNGA3 biosynthesis as a potential mechanism

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

Scalable recombinant adeno-associated virus production using recombinant herpes simplex virus type 1 coinfection of suspension-adapted mammalian cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

CNGB3 mutations account for 50% of all cases with autosomal recessive achromatopsia

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

Mutations in the cone photoreceptor G-protein alpha-subunit gene GNAT2 in patients with achromatopsia

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

CNGA3 mutations in hereditary cone photoreceptor disorders

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

29 September 2017

Impact of preexisting vector immunity on the efficacy of adeno-associated virus-based HIV-1 Gag vaccines.

1 reference

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27 June 2018

Genetic etiology and clinical consequences of complete and incomplete achromatopsia.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

4 September 2018

Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

4 September 2018

Canine CNGB3 mutations establish cone degeneration as orthologous to the human achromatopsia locus ACHM3.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4851180

4 September 2018

Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia

1 reference

https://pubmed.ncbi.nlm.nih.gov/27003752

12 December 2020

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10.1089/HUMC.2015.163

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30 March 2020

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30 March 2020

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30 March 2020

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