| 摘要 |
The present invention provides a method for targeting a particular mRNA sequence in vivo by oral administration of a morpholino antisense compound having uncharged phosphorus-containing backbone linkages. Also disclosed is a non-invasive method of detecting and quantitating the in vivo presence of RNA containing one or more selected target sequences. The method includes administering to a subject a nuclease-resistant antisense oligomer which hybridizes by Watson-Crick base pairing to a region of the target RNA with a Tm substantially greater than 37° C. The oligomer is able to complex intracellularly with target RNA, and is released from intracellular sites as a nuclease-resistant heteroduplex, which can then be measured in a body fluid sample, e.g., urine. |
