In our hands, cholesterol-conjugated antagomirs were not efficacious in inhibiting expression of miRNAs or their target mRNAs following intrathecal delivery.
Thus, the LV-Crym-HA was efficacious to increase Crym expression in the mouse striatum.
Both of these therapies, now on the brink of phase 2 and 3 trials, have proven efficacious in restoring dystrophin expression in the mdx mouse model [ 24].
Recently, exon skipping strategies have proven to be efficacious in restoring functional dystrophin expression in models of muscular dystrophy including the mdx mouse, the GRMD dog and muscle stem cells from DMD patients and in DMD patients by local intramuscular injection [1] [6].
The results in Figure 4 demonstrate that these expression vectors are efficacious.
Vaccination generated an efficacious protective effect, but no expression differences were detected at 2 weeks post vaccination.
In case of explicit redundancy within the IL-10 cytokine family, modulation of their expression could prove more efficacious than targeting the cytokines individually.
Further, it suggests that transcription-repressing drugs would be most efficacious in cancers possessing low BCL2L2 expression.
The knockdown in our hands was efficacious in that the sponge doubled the expression of the inverse reporter, but it did not restore the inverse reporter to the levels seen in cells lacking miR-132.
Taken together, this study provides convincing evidence that a combinational therapeutic treatment regime that modulates Mcl-1 expression levels may be an efficacious approach to sensitize GBM to the apoptosis-inducing effects of TRAIL.
