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Journal article

Codon-optimized RPGR improves stability and efficacy of AAV8 gene therapy in two mouse models of X-linked retinitis pigmentosa

Abstract:

X-linked retinitis pigmentosa (XLRP) is generally a severe form of retinitis pigmentosa, a neurodegenerative, blinding disorder of the retina. 70% of XLRP cases are due to mutations in the retina-specific isoform of the gene encoding retinitis pigmentosa GTPase regulator (RPGRORF15). Despite successful RPGRORF15 gene replacement with adeno-associated viral (AAV) vectors being established in a number of animal models of XLRP, progressio...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1016/j.ymthe.2017.05.005

Authors


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Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Author
ORCID:
0000-0002-6612-6162
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Oxford college:
St Cross College
Role:
Author
More by this author
Role:
Author
ORCID:
0000-0002-8954-8499
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Medical Research Council More from this funder
Publisher:
Cell Press Publisher's website
Journal:
Molecular Therapy Journal website
Volume:
25
Issue:
8
Pages:
1854-1865
Publication date:
2017-08-01
Acceptance date:
2017-05-05
DOI:
EISSN:
1525-0024
ISSN:
1525-0016
Pmid:
28549772

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