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Journal article

Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia

Abstract:

Retinal gene therapy is increasingly recognized as a novel molecular intervention that has huge potential in treating common causes of blindness, the majority of which have a genetic aetiology1,2,3,4,5. Choroideremia is a chronic X-linked retinal degeneration that was first described in 18726. It leads to progressive blindness due to deficiency of Rab-escort protein 1 (REP1). We designed an adeno-associated viral vector to express REP1 and assessed it in a gene therapy clinical trial by subre...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1038/s41591-018-0185-5

Authors


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Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Clinical Neurosciences
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Author
ORCID:
0000-0001-9878-4621
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Clinical Neurosciences
Role:
Author
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Clinical Neurosciences
Role:
Author
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Clinical Neurosciences
Role:
Author
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Health Foundation More from this funder
Lanvern Foundation More from this funder
Fight for Sight More from this funder
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Publisher:
Springer Nature Publisher's website
Journal:
Nature Medicine Journal website
Volume:
24
Pages:
1507–1512
Publication date:
2018-10-08
Acceptance date:
2018-08-06
DOI:
EISSN:
1546-170X
ISSN:
1078-8956
Source identifiers:
926135
Pubs id:
pubs:926135
UUID:
uuid:54465eca-31ec-4541-84ea-4fb93ffb56cf
Local pid:
pubs:926135
Deposit date:
2018-10-10

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