Biomedical Engineering Reference
In-Depth Information
Chapter 6
Bioresponsive Nanoparticles
for the Intracellular Delivery
of RNAi Therapeutics
Kenneth Alan Howard
Department of Molecular Biology and Genetics, Interdisciplinary Nanoscience Center,
University of Aarhus, 8000 Aarhus C, Denmark
kenh@inano.au.dk
The process of RNA interference (RNAi) by which cellular gene
expression is controlled through the specific action of small
interfering RNA (siRNA) is set to revolutionalise disease treatment.
A greater understanding of the RNA pathway has resulted in an
expanding repertoire of potential RNAi-based drugs whose action
is dependent on intracellular target interaction. This work describes
the use of polymeric nanoparticles used to overcome intracellular
barriers and facilitate interaction of RNAi triggers with the RNAi
machinery. Focus will be given to bioresponsive nanoparticles based
on copolypeptides and hyperbranched polymers for controlling the
intracellular spatial and temporal effects of synthetic microRNA and
siRNA.
6.1
Introduction
The discovery that double stranded RNA mediates cellular gene
silencing by the process of RNAi has paved the way towards a new
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