Conclusion
Targeting pathways of human disease with microRNA-based drugs represents a novel and potentially powerful therapeutic approach.
Recent data demonstrate not only that dysregulated microRNAs are associated with and can cause human disease, but that selective
modulation through anti-miR intervention can provide therapeutic benefits. Anti-miR oligonucleotides can be easily administered
through local or parenteral injection routes with sufficient uptake of the agent to achieve sustained target inhibition in
tissues and organs without the need of formulation. Improvements in anti-miR chemical design and pharmacokinetic properties
will allow further exploration of microRNA biology and broaden the utility of microRNA therapeutics.
Kevin Steffy, PhD,* is the global alliance manager, Charles Allerson, PhD, is the associate director of chemistry, and Balkrishen Bhat, PhD, is the senior director of chemistry, all at Regulus Therapeutics, 3545 John Hopkins Ct., San Diego, CA 92121, tel. 858.202.6321,
ksteffy@regulusrx.com
.
*To whom all correspondence should be addressed.
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