Appeal No. 2003-1792
Application No. 09/434,598
Ribozymes and external guide sequences result in cleavage of the fusion transcript,
and antisense RNA and triple helix-forming RNA block expression through hybridization
to a nucleic acid molecule involved in the expression of the fusion transcript. Id.
Inhibitory oligomers are based on affector molecules identified as altering the
expression of an RNA of interest. Specification, page 9.
An example of an external guide sequence is a sequence for promoting cleavage
by eukaryotic RNAse P, and the external guide sequence contains sequences which
are complementary to the target RNA and which forms secondary and tertiary structure
akin to portions of a tRNA molecule. Specification, page 20.
The specification states that an advantage of the “disclosed method is that all, or
a substantial number of accessible sites in the RNA of interest can be determined in
one assay. Such sites, determined to be accessible for one type of affector molecule,
may be accessible for other types of affector molecules. In the case of ribozymes and
external guide sequences, the disclosed method allows assessment not just of
cleavage of the RNA of interest, but also of an ultimate desired phenotype (that is, loss
of the phenotype supported by the RNA of interest) as a result of such cleavage.”
Specification, page 9.
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