siRNA mediated gene silencing continues to hold great potential as a therapeutic modality.  The central challenge in this area remains the ability to deliver siRNA in vivo in a potent and safe manner.  One attractive approach is the use of siRNA conjugated to specific molecules which are designed to facilitate siRNA delivery to the cytosol via either cellular or endolysosomal membrane translocation.  This requires the design of conjugates which possess properties amenable to both hydrophilic and hydrophobic environments.  One potential system is the cholic acid based �molecular umbrella�,  which possess two or more cholic acids attached via lysine linkers.  The dynamic amphiphilic nature of this system allows it to create both a hydrophilic exterior in a hydrophilic environment and a hydrophobic exterior in a hydrophobic environment.  This work will detail the design and application of �molecular umbrella� conjugates of siRNA for in vitro and in vivo delivery.

Molecular umbrella conjugates for the in vivo delivery of siRNA

246th National Meeting (2013)

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