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Gene Therapy for Choroideremia Using an Adeno-Associated Viral (AAV) Vector.

Abstract:

Choroideremia is an outer retinal degeneration with a characteristic clinical appearance that was first described in the nineteenth century. The disorder begins with reduction of night vision and gradually progresses to blindness by middle age. The appearance of the fundus in sufferers is recognizable by the characteristic pale color caused by the loss of the outer retina, retinal-pigmented epithelium, and choroidal vessels, leading to exposure of the underlying sclera. Choroideremia shows X-...

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Publisher copy:
10.1101/cshperspect.a017293

Authors


More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, Clinical Neuroscience
More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, Clinical Neuroscience
More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, Clinical Neuroscience, Biomedical Research Centre
Journal:
Cold Spring Harbor perspectives in medicine
Publication date:
2014-10-05
DOI:
EISSN:
2157-1422
URN:
uuid:8aec8d54-24cb-4e0d-a9a8-cdfcad0d8bd1
Source identifiers:
488429
Local pid:
pubs:488429
Language:
English

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