Lipid-Like Delivery Materials for Efficient siRNA Delivery - Nanotechnology for the Delivery of Therapeutic Nucleic Acids - page 153

Chapter 7

Lipid-Like Delivery Materials for
Efficient siRNA Delivery

James Dahlman, a,b Robert Langer, a,b and Michael Goldberg c, *
a

Harvard-MIT Division of Health Science and Technology,
Cambridge, MA, 02139, USA
b

David H. Koch Center for Integrative Cancer Research, MIT,
Cambridge, MA, 02139, USA
c

Dana-Farber Cancer Institute, Boston, MA, 02215, USA
* [e-mail]

Realizing the full potential of RNAi therapeutics will require the
advent of safe and efficient carriers. Since the structure of the ideal
vehicle remains unknown, the ability to generate and evaluate many
compounds in a high-throughput manner is likely to direct our
efforts. One-step conjugation chemistries facilitate the synthesis of
large libraries, whose members can be screened for efficacy both in

vitro . Comparison of the top-performing compounds can

yield structure-function relationships that can, in turn, inform the
synthesis of iterative libraries. This chapter describes the synthesis,
screening, formulation, evolution, and application of “lipidoids”,
a novel class of lipid-like molecules that highlights the utility of
combinatorial approaches for the production of effective siRNA
delivery vehicles.

and in vivo