Journal article
A fragmented adeno-associated viral dual vector strategy for treatment of diseases caused by mutations in large genes leads to expression of hybrid transcripts
- Abstract:
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Objective
Dual vector AAV systems are being utilised to enable gene therapy for disorders in which the disease gene is too large to ft into a single capsid. Fragmented adeno-associated viral (fAAV) vectors containing single inverted terminal repeat truncated transgenes have been considered as one such gene replacement strategy. Here we aim to add to the current understanding of the molecular mechanisms employed by fAAV dual vector systems.
Methods
Oversized (>...
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- Publication status:
- Published
- Peer review status:
- Peer reviewed
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(Version of record, pdf, 1.2MB)
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- Publisher copy:
- 10.4172/2157-7412.1000311
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Authors
Funding
Medical Research Council
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Fight for Sight
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Royal College of Surgeons of Edinburgh
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Marie Curie Foundation
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Knoop Fellowship
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Bibliographic Details
- Publisher:
- OMICS International Publisher's website
- Journal:
- Journal of Genetic Syndromes & Gene Therapy Journal website
- Volume:
- 7
- Pages:
- 311
- Publication date:
- 2016-11-01
- Acceptance date:
- 2016-11-07
- DOI:
- ISSN:
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2157-7412
- Source identifiers:
-
672276
Item Description
- Keywords:
- Pubs id:
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pubs:672276
- UUID:
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uuid:baa37546-e286-4687-a1c4-5f5e42905c88
- Local pid:
- pubs:672276
- Deposit date:
- 2017-01-22
Terms of use
- Copyright holder:
- McClements et al
- Copyright date:
- 2016
- Notes:
- © 2016 McClements ME, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited
- Licence:
- CC Attribution (CC BY)
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