AI Article Synopsis
- The study focuses on using RNA antagonists and gene therapy to modulate specific gene targets effectively.
- Detection of tissue-specific mRNAs in biological fluids, like serum and cerebrospinal fluid, indicates the potential for monitoring gene expression changes.
- A noninvasive method for tracking RNA modulation could enhance the development of RNA-based treatments in clinical settings.
Article 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.
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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3895267 | PMC |
| http://dx.doi.org/10.1261/rna.042507.113 | DOI Listing |
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