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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:

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.

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

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Publisher copy:
10.4172/2157-7412.1000311

Authors


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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
Medical Research Council More from this funder
Fight for Sight More from this funder
Royal College of Surgeons of Edinburgh More from this funder
Knoop Fellowship More from this funder
Marie Curie Foundation More from this funder
Publisher:
OMICS International Publisher's website
Journal:
Journal of Genetic Syndromes & Gene Therapy Journal website
Volume:
7
Pages:
311
Publication date:
2016-11-05
Acceptance date:
2016-11-07
DOI:
ISSN:
2157-7412
Pubs id:
pubs:672276
URN:
uri:baa37546-e286-4687-a1c4-5f5e42905c88
UUID:
uuid:baa37546-e286-4687-a1c4-5f5e42905c88
Local pid:
pubs:672276

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