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Inclusion of the woodchuck hepatitis virus posttranscriptional regulatory element enhances AAV2-driven transduction of mouse and human retina

Abstract:

The woodchuck hepatitis virus posttranscriptional regulatory element (WPRE) has been included in the transgene cassette of adeno-associated virus (AAV) in several gene therapy clinical trials, including those for inherited retinal diseases. However, the extent to which WPRE increases transgene expression in the retina is still unclear. To address this question, AAV2 vectors containing a reporter gene with and without WPRE were initially compared in vitro and subsequently in vivo by subretinal...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Publisher's version

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Publisher copy:
10.1016/j.omtn.2016.12.006

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Department:
Oxford, MSD, Clinical Neurosciences
Role:
Author
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Department:
Oxford, MSD, Clinical Neurosciences
Role:
Author
More by this author
Department:
Oxford, MSD, Clinical Neurosciences
Role:
Author
More by this author
Department:
Oxford, MSD, Clinical Neurosciences
Role:
Author
More by this author
Department:
Oxford, MSD, Clinical Neurosciences
Role:
Author
Fight for Sight More from this funder
Health Foundation More from this funder
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Grant:
MR/K007629/1, MR/K003690/1, MR/N00101X/1, MR/K007629/1
Royal College of Surgeons of Edinburgh More from this funder
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Publisher:
American Society of Gene and Cell Therapy Publisher's website
Journal:
Molecular Therapy - Nucleic Acids Journal website
Volume:
6
Pages:
198-208
Publication date:
2017-03-05
DOI:
ISSN:
2162-2531
Pubs id:
pubs:681401
URN:
uri:53988a61-7344-4bf5-a9c2-4834697c7426
UUID:
uuid:53988a61-7344-4bf5-a9c2-4834697c7426
Local pid:
pubs:681401

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