Tissue-specific gene silencing monitored in circulating RNA
Name
Sehgal-2014-Tissue-specific gene.pdf
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516.23 KB
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Author(s)
Sehgal, Alfica
Chen, Qingmin
Gibbings, Derrick
Sah, Dinah W. Y.
Bumcrot, David A.
Date Issued
December 2013
Journal
RNA
Publisher
Cold Spring Harbor Laboratory Press
Citation
Sehgal, A., Q. Chen, D. Gibbings, D. W. Y. Sah, and D. Bumcrot. “Tissue-Specific Gene Silencing Monitored in Circulating RNA.” RNA 20, no. 2 (February 1, 2014): 143–149.
Version
Final published version
Abstract
Pharmacologic target gene modulation is the primary objective for RNA antagonist strategies and gene therapy. Here we show that mRNAs encoding tissue-specific gene transcripts can be detected in biological fluids and that RNAi-mediated target gene silencing in the liver and brain results in quantitative reductions in serum and cerebrospinal fluid mRNA levels, respectively. Further, administration of an anti-miRNA oligonucleotide resulted in decreased levels of the miRNA in circulation. Moreover, ectopic expression of an adenoviral transgene in the liver was quantified based on measurement of serum mRNA levels. This noninvasive method for monitoring tissue-specific RNA modulation could greatly advance the clinical development of RNA-based therapeutics.
MIT Department
Koch Institute for Integrative Cancer Research at MIT
Terms of Use
Creative Commons Attribution-Noncommerical
Persistent DSpace Link
DOI of Published Version
https://doi.org/10.1261/rna.042507.113
