Journal article

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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APA Style

Edwards, T., Jolly, J., Groppe, M., Barnard, A., Cottriall, C., Tolmachova, T., Black, G., Webster, A., Lotery, A., Holder, G., Xue, K., Downes, S., Simunovic, M., Seabra, M., & MacLaren, R. (2016). Visual acuity after retinal gene therapy for Choroideremia. New England Journal of Medicine, 374(20), 1996–1998.

MLA Style

Edwards, T., et al. “Visual Acuity after Retinal Gene Therapy for Choroideremia.” New England Journal of Medicine, vol. 374, no. 20, Massachusetts Medical Society, 2016, pp. 1996–98.

Chicago Style

Edwards, T, J Jolly, M Groppe, A Barnard, C Cottriall, T Tolmachova, G Black, et al. 2016. “Visual Acuity after Retinal Gene Therapy for Choroideremia.” New England Journal of Medicine 374 (20): 1996–98.
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Files:: NEJMc1509501CHM.pdf

(Preview, Accepted manuscript, pdf, 287.6KB, Terms of use)

Publisher copy:: 10.1056/NEJMc1509501

Authors

+ Edwards, T More by this author

Institution:: University of Oxford
Division:: MSD
Department:: Clinical Neurosciences
Role:: Author

+ Jolly, J More by this author

Institution:: University of Oxford
Division:: MSD
Department:: Clinical Neurosciences
Role:: Author

+ Groppe, M More by this author

Institution:: University of Oxford
Division:: MSD
Department:: Clinical Neurosciences
Role:: Author

+ Barnard, A More by this author

Institution:: University of Oxford
Division:: MSD
Department:: Clinical Neurosciences
Role:: Author

+ Cottriall, C More by this author

Role:: Author

More authors...

+ Wellcome Trust More from this funder

Grant:: HICF-1009-006) Health Innovation Challenge Fund

+ Department of Health More from this funder

Grant:: HICF-1009-006) Health Innovation Challenge Fund

+ Choroideremia Research Foundation More from this funder

+ Royal College of Surgeons of Edinburgh More from this funder

+ NIHR Oxford Biomedical Research Centre More from this funder

More funders...

Publisher:: Massachusetts Medical Society
Journal:: New England Journal of Medicine More from this journal
Volume:: 374
Issue:: 20
Pages:: 1996-1998
Publication date:: 2016-05-19
Acceptance date:: 2016-04-27
DOI:: 10.1056/NEJMc1509501
EISSN:: 1533-4406
ISSN:: 0028-4793

Language:: English
Pubs id:: pubs:619334
UUID:: uuid:94f8e98a-f0bb-4106-8d86-d42cf9cba0a2
Local pid:: pubs:619334
Source identifiers:: 619334
Deposit date:: 2016-06-03

Terms of use

Copyright holder:: Massachusetts Medical Society
Copyright date:: 2016
Notes:: Copyright © 2016 Massachusetts Medical Society. All rights reserved.

Licence:: Terms and Conditions of Use for Oxford University Research Archive

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