(Q24627838)

22 June 2017

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

1 reference

Jennifer Wellman McDonnell

22 June 2017

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0 references

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9

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19

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Enrico M. Surace

Enrico M Surace

10

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Francesco Testa

Francesco Testa

3

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Francesca Simonelli

Francesca Simonelli

1

0 references

Eric A Pierce

Eric A Pierce

4

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Sandro Banfi

Sandro Banfi

9

0 references

Alberto Auricchio

Alberto Auricchio

22

0 references

Settimio Rossi

7

Settimio Rossi

1 reference

22 June 2017

2

Albert M Maguire

1 reference

22 June 2017

8

Kathleen Marshall

1 reference

22 June 2017

5

Federico Mingozzi

1 reference

22 June 2017

Jeannette L Bennicelli

6

1 reference

22 June 2017

Junwei Sun

11

1 reference

22 June 2017

T Michael Redmond

12

1 reference

22 June 2017

Xiaosong Zhu

13

1 reference

22 June 2017

Kenneth S Shindler

14

1 reference

22 June 2017

Gui-Shuang Ying

15

1 reference

22 June 2017

Carmela Ziviello

16

1 reference

22 June 2017

Carmela Acerra

17

1 reference

22 June 2017

J Fraser Wright

18

1 reference

22 June 2017

Katherine A High

20

1 reference

22 June 2017

Jean Bennett

21

1 reference

22 June 2017

Jennifer Wellman McDonnell

19

1 reference

22 June 2017

language of work or name

English

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publication date

1 December 2009

1 reference

22 June 2017

1 reference

22 June 2017

18

1 reference

22 June 2017

643-650

1 reference

22 June 2017

3

1 reference

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

22 June 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

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

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

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

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

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

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Mutations in SPATA7 cause Leber congenital amaurosis and juvenile retinitis pigmentosa

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

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

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Leber congenital amaurosis: genes, proteins and disease mechanisms

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

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

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

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

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Impairment of the transient pupillary light reflex in Rpe65(-/-) mice and humans with leber congenital amaurosis

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Molecular genetics of Leber congenital amaurosis

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Stargardt's Disease and Fundus Flavimaculatus

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Standard for clinical electroretinography (2004 update).

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Gene therapy restores vision in a canine model of childhood blindness

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2839440

Periodic alternating nystagmus clearing after vitrectomy

28 November 2018

1 reference

12 December 2020

Variability of visual field measurements in normal subjects and patients with retinitis pigmentosa

1 reference

12 December 2020

Changes in nystagmus after simultaneous surgery for bilateral congenital cataracts

1 reference

12 December 2020

Variability of the relative afferent pupillary defect

1 reference

12 December 2020

Vision in Leber congenital amaurosis

1 reference

12 December 2020

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

1 reference

12 December 2020

Identifiers

DOI

10.1038/MT.2009.277

1 reference

OpenCitations bibliographic resource ID

22 June 2017

1288215

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

1 reference