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Development of a Positive-readout Mouse Model of siRNA Pharmacodynamics.

Abstract:

Development of RNAi-based therapeutics has the potential to revolutionize treatment options for a range of human diseases. However, as with gene therapy, a major barrier to progress is the lack of methods to achieve and measure efficient delivery for systemic administration. We have developed a positive-readout pharmacodynamic transgenic reporter mouse model allowing noninvasive real-time assessment of siRNA activity. The model combines a luciferase reporter gene under the control of regulato...

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Publication status:
Published

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Publisher copy:
10.1038/mtna.2013.63

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Publisher:
Nature Publishing Group
Journal:
Molecular therapy. Nucleic acids
Volume:
2
Issue:
NOV
Pages:
e133
Publication date:
2013-01-01
DOI:
EISSN:
2162-2531
ISSN:
2162-2531
Source identifiers:
440276
Language:
English
Keywords:
Pubs id:
pubs:440276
UUID:
uuid:3aa50685-14c0-4270-86cf-577941daf749
Local pid:
pubs:440276
Deposit date:
2013-12-12

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