Journal article icon

Journal article

Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4-/- mouse and bipolar cells in the rd1 mouse and human retina ex-vivo

Abstract:

Gene therapy using adeno-associated viral vectors (AAV) for the treatment of retinal degenerations has shown safety and efficacy in clinical trials. However, very high levels of vector expression may be necessary for the treatment of conditions such as Stargardt disease where a dual vector approach is potentially needed, or in optogenetic strategies for end-stage degeneration in order to achieve maximal light sensitivity. In this study, we assessed two vectors with single capsid mutations, rA...

Expand abstract
Publication status:
Published
Peer review status:
Peer reviewed

Actions


Access Document


Files:
Publisher copy:
10.1038/gt.2016.54

Authors


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
Royal College of Surgeons More from this funder
Fight for Sight More from this funder
Medical Research Council More from this funder
Wellcome Trust More from this funder
National Institute for Health Research Oxford Biomedical Research Centres More from this funder
Publisher:
Nature Publishing Group Publisher's website
Journal:
Gene Therapy Journal website
Volume:
23
Pages:
767–774
Publication date:
2016-08-11
Acceptance date:
2016-04-20
DOI:
EISSN:
1476-5462
ISSN:
0969-7128
Source identifiers:
627019
Pubs id:
pubs:627019
UUID:
uuid:940b24a3-d587-4432-ac96-02224c8a3ae2
Local pid:
pubs:627019
Deposit date:
2016-06-09

Terms of use


Views and Downloads






If you are the owner of this record, you can report an update to it here: Report update to this record

TO TOP