Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration (Q24627838)

22 June 2017

title

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

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

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Eric A Pierce

Eric A Pierce

4

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Sandro Banfi

Sandro Banfi

9

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Alberto Auricchio

Alberto Auricchio

22

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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

author name string

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

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22 June 2017

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22 June 2017

volume

18

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22 June 2017

issue

3

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22 June 2017

page(s)

643-650

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

22 June 2017

cites work

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

1288215

1 reference