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Full-Text Articles in Physical Sciences and Mathematics
Designed Guanidinium-Rich Amphipathic Oligocarbonate Molecular Transporters Complex, Deliver And Release Sirna In Cells, E. I. Geihe, Christina B. Cooley, J. R. Simon, M. K. Kiesewetter, J. A. Edward, R. P. Hickerson, R. L. Kaspar, J. L. Hedrick, R. M. Waymouth, P. A. Wender
Designed Guanidinium-Rich Amphipathic Oligocarbonate Molecular Transporters Complex, Deliver And Release Sirna In Cells, E. I. Geihe, Christina B. Cooley, J. R. Simon, M. K. Kiesewetter, J. A. Edward, R. P. Hickerson, R. L. Kaspar, J. L. Hedrick, R. M. Waymouth, P. A. Wender
Christina B Cooley
The polyanionic nature of oligonucleotides and their enzymatic degradation present challenges for the use of siRNA in research and therapy; among the most notable of these is clinically relevant delivery into cells. To address this problem, we designed and synthesized the first members of a new class of guanidinium-rich amphipathic oligocarbonates that noncovalently complex, deliver, and release siRNA in cells, resulting in robust knockdown of target protein synthesis in vitro as determined using a dual-reporter system. The organocatalytic oligomerization used to synthesize these co-oligomers is step-economical and broadly tunable, affording an exceptionally quick strategy to explore chemical space for optimal …