penetrance of the molecule and therefore can achieve selective gene knockdown in vivo (86). This
approach has recently been used to inhibit expression of an oncogene in a glioblastoma model
using small interfering RNAs (87).
Theoretically, either SNAs or ASOs could be used to selectively target components of endoge-
nous DNA methylation machinery in the CNS. Although this general category of tool has been
available to researchers for many years in the form of virally mediated gene knockdown, these
approaches confer a superior pharmacology: ease of entrance into cells without viral packaging
or transfection reagents, stability of the ASO or SNA over time, and potentially prolonged gene
knockdown with a single drug dose. Furthermore, the one-size-fits-all delivery method incumbent
to both of these techniques is rapidly adaptable to new gene targets and could even support multi-
plexed targeting of several genes at the same time. Although the ability to target these constructs
to specific brain locations within human populations using intrathecal or intracerebroventricular
injections remains a challenge for pharmacotherapy, there are promising developments poised to
solve this problem (88).
penetrance of the molecule and therefore can achieve selective gene knockdown in vivo (86). Thisapproach has recently been used to inhibit expression of an oncogene in a glioblastoma modelusing small interfering RNAs (87).Theoretically, either SNAs or ASOs could be used to selectively target components of endoge-nous DNA methylation machinery in the CNS. Although this general category of tool has beenavailable to researchers for many years in the form of virally mediated gene knockdown, theseapproaches confer a superior pharmacology: ease of entrance into cells without viral packagingor transfection reagents, stability of the ASO or SNA over time, and potentially prolonged geneknockdown with a single drug dose. Furthermore, the one-size-fits-all delivery method incumbentto both of these techniques is rapidly adaptable to new gene targets and could even support multi-plexed targeting of several genes at the same time. Although the ability to target these constructsto specific brain locations within human populations using intrathecal or intracerebroventricularinjections remains a challenge for pharmacotherapy, there are promising developments poised tosolve this problem (88).
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