| 摘要 |
<p>Synthetic small double stranded RNAs that can induce gene specific inhibition of expression are provided, as are methods for selecting and constructing optimal small double-stranded RNAs. Provided RNAs are suitable for interference or inhibition of expression of a target gene and comprise double stranded RNAs of about to about 40 nucleotides containing a 3' and/or 5' overhang on each strand having a length of O-nucleotide to 5-nucleotides, wherein the sequence of the double stranded RNAs are substantially identical (e.g., differing by no more than 30%, or more preferably no more than 10% ) to a portion of a mRNA or transcript of the target gene for which interference or inhibition of expression is desired. The current disclosure is also directed to the use of such synthetic small double stranded RNAs as reverse genetic and/or therapeutic tools. Also provided are methods, including high-throughput methods, of testing, analyzing, and optimizing short double-stranded 15 RNAs for use in gene specific inhibition of expression.</p> |
