RNA interference, a technique wherein double stranded, short interfering RNA (siRNA) is used to destroy mRNA before it is translated, holds promise for treating many diseases that are otherwise �undruggable� with current technology.  However, the widespread use of siRNA for disease prevention and treatment requires the development of clinically suitable, safe and effective drug delivery vehicles.  We present the synthesis and evaluation of a microparticulate gene delivery system based on acid-sensitive acetal-modified dextran (Ac-DEX).  Due to their biocompatibility, biodegradability, and tunability, Ac-DEX-based particles have the potential to be used in a wide range of gene delivery applications.  Modification of Ac-DEX facilitates the preparation of siRNA-loaded particles capable of efficiently transfecting non-phagocytic cells with minimal toxicity.  We are currently investigating the effect of size and particle degradation rate on delivery efficiency as well as the modification of these particles with peptide targeting ligands.

Acetal-modified dextran particles for siRNA delivery

239th National Meeting (2010)

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