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Journal article

Ocular gene therapy for choroideremia: clinical trials and future perspectives

Abstract:
Introduction: Gene therapy offers the potential for targeted replacement of single gene defects in inherited retinal degenerations. Areas covered: Choroideremia is an X-linked blinding retinal disease resulting from deficiency of the CHM gene product, REP1. The disease represents an ideal target for retinal gene therapy, as it is readily diagnosed in the clinic, relatively homogenous in phenotype and slow progressing, thereby providing a wide therapeutic win... Expand abstract
Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1080/17469899.2018.1475232

Authors


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Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Author
ORCID:
0000-0003-0065-9131
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Clinical Neurosciences
Oxford college:
Merton College
Role:
Author
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Grant:
MechanismEvaluation(EME)Award
Efficacy
NIHR Oxford Biomedical Research Centre More from this funder
Publisher:
Taylor and Francis Publisher's website
Journal:
Expert Review of Ophthalmology Journal website
Volume:
13
Issue:
3
Pages:
129-138
Publication date:
2018-05-18
Acceptance date:
2018-05-08
DOI:
EISSN:
1746-9902
ISSN:
1746-9899
Source identifiers:
911653
Keywords:
Pubs id:
pubs:911653
UUID:
uuid:b8ecd08b-1d80-45d2-9219-e419ae8cb1f9
Local pid:
pubs:911653
Deposit date:
2018-10-15

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