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Visual acuity after retinal gene therapy for choroideremia

Visual acuity after retinal gene therapy for choroideremia
Visual acuity after retinal gene therapy for choroideremia
To the Editor: Two recent clinical reports of retinal gene therapy with adeno-associated virus (AAV) vectors in patients with Leber's congenital amaurosis showed initial gains in visual function that subsequently declined.(1),(2) We previously reported early improvement in visual acuity in two of six patients who received retinal gene therapy in one eye (the study eye) to treat choroideremia,(3) a disease that is characterized by atrophy of the choriocapillaris and retinal pigment epithelium and involves vision loss that leads to blindness. Choroideremia is caused by loss-of-function mutations in the gene CHM. We delivered nonmutated CHM in an AAV vector (AAV.REP1) . . .
1996-1998
Edwards, Thomas L.
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Jolly, Jasleen K.
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Groppe, Markus
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Barnard, Alun R.
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Cottriall, Charles L.
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Tolmachova, Tanya
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Black, Graeme C.
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Webster, Andrew R.
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Lotery, Andrew J.
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Holder, Graham E.
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Xue, Kanmin
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Downes, Susan M.
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Simunovic, Matthew P.
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Seabra, Miguel C.
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MacLaren, Robert E.
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Edwards, Thomas L.
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Jolly, Jasleen K.
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Groppe, Markus
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Barnard, Alun R.
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Cottriall, Charles L.
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Tolmachova, Tanya
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Black, Graeme C.
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Webster, Andrew R.
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Lotery, Andrew J.
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Holder, Graham E.
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Xue, Kanmin
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Downes, Susan M.
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Simunovic, Matthew P.
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Seabra, Miguel C.
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MacLaren, Robert E.
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Edwards, Thomas L., Jolly, Jasleen K., Groppe, Markus, Barnard, Alun R., Cottriall, Charles L., Tolmachova, Tanya, Black, Graeme C., Webster, Andrew R., Lotery, Andrew J., Holder, Graham E., Xue, Kanmin, Downes, Susan M., Simunovic, Matthew P., Seabra, Miguel C. and MacLaren, Robert E. (2016) Visual acuity after retinal gene therapy for choroideremia. New England Journal of Medicine, 374 (20), 1996-1998. (doi:10.1056/NEJMc1509501). (PMID:27120491)

Record type: Letter

Abstract

To the Editor: Two recent clinical reports of retinal gene therapy with adeno-associated virus (AAV) vectors in patients with Leber's congenital amaurosis showed initial gains in visual function that subsequently declined.(1),(2) We previously reported early improvement in visual acuity in two of six patients who received retinal gene therapy in one eye (the study eye) to treat choroideremia,(3) a disease that is characterized by atrophy of the choriocapillaris and retinal pigment epithelium and involves vision loss that leads to blindness. Choroideremia is caused by loss-of-function mutations in the gene CHM. We delivered nonmutated CHM in an AAV vector (AAV.REP1) . . .

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e-pub ahead of print date: 27 April 2016
Published date: 19 May 2016
Organisations: Clinical & Experimental Sciences

Identifiers

Local EPrints ID: 393724
URI: https://eprints.soton.ac.uk/id/eprint/393724
PURE UUID: 443a5a0a-2db8-4d23-8a36-f39664b7fc63
ORCID for Andrew J. Lotery: ORCID iD orcid.org/0000-0001-5541-4305

Catalogue record

Date deposited: 03 May 2016 13:25
Last modified: 10 Dec 2019 01:46

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Contributors

Author: Thomas L. Edwards
Author: Jasleen K. Jolly
Author: Markus Groppe
Author: Alun R. Barnard
Author: Charles L. Cottriall
Author: Tanya Tolmachova
Author: Graeme C. Black
Author: Andrew R. Webster
Author: Graham E. Holder
Author: Kanmin Xue
Author: Susan M. Downes
Author: Matthew P. Simunovic
Author: Miguel C. Seabra
Author: Robert E. MacLaren

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