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...
Expand abstract
- Publication status:
- Published
- Peer review status:
- Peer reviewed
Actions
Access Document
- Files:
-
-
(Version of record, 2.5MB)
-
- Publisher copy:
- 10.1016/j.ymthe.2017.05.005
Why is the content I wish to access not available via ORA?
Bibliographic data (the information relating to research outputs) and full-text items (e.g. articles, theses, reports, etc.) arrive in ORA from several different sources. Unfortunately we are not able to make available the full-text for every research output.
Please contact the ORA team (
) if you have queries regarding unavailable content OR if you are aware of a full-text copy we can make available.
) if you have queries regarding unavailable content OR if you are aware of a full-text copy we can make available.
Content may be unavailable for the following four reasons
-
Version unsuitable
-
We have not obtained a suitable full-text for a given research output. See this page for more information.
-
Recently completed
-
Sometimes content is held in ORA but is unavailable for a fixed period of time to comply with the policies and wishes of rights holders.
-
Permissions
-
All content made available in ORA should comply with relevant rights, such as copyright. See this page for more information.
-
Clearance
-
Some thesis volumes scanned as part of the digitisation scheme funded by Dr Leonard Polonsky are currently unavailable due to sensitive material or uncleared third-party copyright content. We are attempting to contact authors whose theses are affected.
Alternative access to the full-text
You may be able to access the full-text directly from the publisher's website using the 'Publisher Copy' link in the 'Links & Downloads' box from a research output's ORA record page. This method may require an institutional or individual subscription to the journal/resource.
Authors
Funding
Medical Research Council
More from this funder
Bibliographic Details
- 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
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
697586
- Local pid:
- pubs:697586
- Deposit date:
- 2021-08-22
Terms of use
- Copyright holder:
- Fischer et al.
- Copyright date:
- 2017
- Rights statement:
- Copyright 2017 The Authors. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).
- Licence:
- CC Attribution (CC BY)
If you are the owner of this record, you can report an update to it here: Report update to this record