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Visual acuity after retinal gene therapy for Choroideremia

Abstract:
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. 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, a disease that is characterized by atrophy of the choriocapillaris and retinal pigment epithelium and involves vision loss that leads to blindness.
Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1056/NEJMc1509501

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Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Author
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More from this funder
Grant:
HICF-1009-006) Health Innovation Challenge Fund
More from this funder
Grant:
HICF-1009-006) Health Innovation Challenge Fund
The Health Foundation More from this funder
The Lanvern Foundation More from this funder
Fight for Sight More from this funder
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Publisher:
Massachusetts Medical Society Publisher's website
Journal:
New England Journal of Medicine Journal website
Volume:
374
Issue:
20
Pages:
1996-1998
Publication date:
2016-05-19
Acceptance date:
2016-04-27
DOI:
EISSN:
1533-4406
ISSN:
0028-4793
Source identifiers:
619334
Language:
English
Pubs id:
pubs:619334
UUID:
uuid:94f8e98a-f0bb-4106-8d86-d42cf9cba0a2
Local pid:
pubs:619334
Deposit date:
2016-06-03

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