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Knockdown and replacement therapy mediated by artificial mirtrons in spinocerebellar ataxia 7

Abstract:

We evaluate a knockdown-replacement strategy mediated by mirtrons as an alternative to allele-specific silencing using spinocerebellar ataxia 7 (SCA7) as a model. Mirtrons are introns that form pre-microRNA hairpins after splicing, producing RNAi effectors not processed by Drosha. Mirtron mimics may therefore avoid saturation of the canonical processing pathway. This method combines gene silencing mediated by an artificial mirtron with delivery of a functional copy of the gene such that both ...

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

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Publisher copy:
10.1093/nar/gkx483

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Department:
Oxford, MSD, Physiology Anatomy & Genetics
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Department:
Oxford, MSD, Physiology Anatomy & Genetics
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Department:
Oxford, MSD, Physiology Anatomy & Genetics
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Publisher:
Oxford University Press Publisher's website
Journal:
Nucleic Acids Research Journal website
Volume:
45
Issue:
13
Pages:
7870-7885
Publication date:
2017-05-30
Acceptance date:
2017-05-26
DOI:
EISSN:
1362-4962
ISSN:
0305-1048
Pubs id:
pubs:698685
URN:
uri:bff0c273-880b-4496-94fe-8aaa31b60ecf
UUID:
uuid:bff0c273-880b-4496-94fe-8aaa31b60ecf
Local pid:
pubs:698685
Language:
English
Keywords:

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