Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer (Q33315634)

25 July 2017

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

1 reference

Leber congenital amaurosis

25 July 2017

0 references

1 reference

19

Albert M Maguire

1 reference

25 July 2017

7

Federico Mingozzi

1 reference

25 July 2017

Jeannette Bennicelli

1

1 reference

25 July 2017

John Fraser Wright

2

1 reference

25 July 2017

Andras Komaromy

3

1 reference

25 July 2017

Jonathan B Jacobs

4

1 reference

25 July 2017

Bernd Hauck

5

1 reference

25 July 2017

Olga Zelenaia

6

1 reference

25 July 2017

Daniel Hui

8

1 reference

25 July 2017

Daniel Chung

9

1 reference

25 July 2017

Tonia S Rex

10

1 reference

25 July 2017

Zhangyong Wei

11

1 reference

25 July 2017

Guang Qu

12

1 reference

25 July 2017

Shangzhen Zhou

13

1 reference

25 July 2017

Caroline Zeiss

14

1 reference

25 July 2017

Valder R Arruda

15

1 reference

25 July 2017

Gregory M Acland

16

1 reference

25 July 2017

Lou F Dell'Osso

17

1 reference

25 July 2017

Katherine A High

18

1 reference

25 July 2017

Jean Bennett

20

1 reference

25 July 2017

22 January 2008

1 reference

25 July 2017

1 reference

25 July 2017

16

1 reference

25 July 2017

3

1 reference

25 July 2017

458-465

1 reference

Mutations in LCA5, encoding the ciliary protein lebercilin, cause Leber congenital amaurosis

25 July 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Hypoglycemia leads to age-related loss of vision

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Independent visual threshold measurements in the two eyes of freely moving rats and mice using a virtual-reality optokinetic system.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65-/- knockout mouse eye results in limited rescue

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Leber congenital amaurosis: comprehensive survey of the genetic heterogeneity, refinement of the clinical definition, and genotype-phenotype correlations as a strategy for molecular diagnosis

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Vitamin A metabolism in the retinal pigment epithelium: genes, mutations, and diseases

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

In vivo gene therapy in young and adult RPE65-/- dogs produces long-term visual improvement.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Molecular genetics of Leber congenital amaurosis

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Retinal dystrophy of Swedish briard/briard-beagle dogs is due to a 4-bp deletion in RPE65.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA).

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

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

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Gene transfer in the RPE65 null mutation dog: relationship between construct volume, visual behavior and electroretinographic (ERG) results

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Gene therapy restores vision in a canine model of childhood blindness

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Cross-species comparison of in vivo reporter gene expression after recombinant adeno-associated virus-mediated retinal transduction

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Real-time, noninvasive in vivo assessment of adeno-associated virus-mediated retinal transduction

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2842085

Recovery phase of the murine rod photoresponse reconstructed from electroretinographic recordings

31 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/18209734

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

UV- and midwave-sensitive cone-driven retinal responses of the mouse: a possible phenotype for coexpression of cone photopigments

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/18209734

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Genetic analysis of RPE65: from human disease to mouse model

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/18209734

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/SJ.MT.6300389

1 reference

25 July 2017

1 reference

25 July 2017

1 reference