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Systemic delivery of MicroRNA using recombinant adeno-associated virus serotype 9 to treat neuromuscular diseases in rodents

Abstract:

RNA interference via the endogenous miRNA pathway regulates gene expression by controlling protein synthesis through post-transcriptional gene silencing. In recent years, miRNA-mediated gene regulation has shown potential for treatment of neurological disorders caused by a toxic gain of function mechanism. However, efficient delivery to target tissues has limited its application. Here we used a transgenic mouse model for spinal and bulbar muscular atrophy (SBMA), a neuromuscular disease cause...

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

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Publisher copy:
10.3791/55724

Authors


Pourshafie, N More by this author
Harmison, GG More by this author
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Publisher:
Journal of Visualized Experiments Publisher's website
Journal:
Journal of Visualized Experiments Journal website
Volume:
2018
Issue:
138
Publication date:
2018-10-08
DOI:
EISSN:
1940-087X
Pubs id:
pubs:912488
URN:
uri:3c579afe-5604-4f77-88b5-723c961d01d2
UUID:
uuid:3c579afe-5604-4f77-88b5-723c961d01d2
Local pid:
pubs:912488
Language:
English

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