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Drug delivery

Cyclodextrin-modified dendritic polyamine (DexAM) construct for delivery of stem cell differentiation factors

DexAM constructs that simultaneously deliver small interfering RNA and small molecules could be used to control stem cell differentiation. The dendritic polyamine portion of the construct is in complex with a negatively charged siRNA, whereas the cyclodextrin portion of the construct is loaded with a hydrophobic small molecule. In neuronal stem cells, DexAM carrying the small molecule retinoic acid and siRNA targeting SRY (sex determining region Y)-box 9 (SOX9) led to about 71% of the stem cells differentiating into neurons, whereas DexAM carrying either component alone achieved about 50% differentiation. Next steps include testing the delivery vehicle in other types of stem cells and in animal models.

SciBX 6(43); doi:10.1038/scibx.2013.1240
Published online Nov. 7, 2013

Patent application filed; unavailable for licensing

Shah, S. et al. J. Am. Chem. Soc.; published online Oct. 9, 2013;
doi:10.1021/ja4071738
Contact: Ki-Bum Lee, Rutgers University, Piscataway, N.J.
e-mail:

kblee@rutgers.edu