ABSTRACT
The use of short interfering ribonucleic acid (siRNA) to induce
RNA interference (RNAi) and to silence gene expression is well
established in basic research and widely used in biological research.
Its high potential for new therapeutic strategies stimulated many
groups and companies worldwide to develop siRNA therapies for
diseases like cancer, viral infections, and chronic diseases. These
efforts revealed, however, that siRNA could only be applied to
patients once several technical and systemic problems are solved:
(1) siRNA stability and the prevention of its degradation have to
be improved, (2) efficient siRNA delivery and gene silencing are
still a major hurdle in vivo, (3) unspecific stimulation of immune
response by siRNA has to be reduced, (4) the balance between target
sequence specificity and the risk of escape mutations has to be
addressed, (5) siRNA production for therapeutic applications has
to be up-scaled, and (6) the cell specificity of RNAi for high-impact
therapeutic strategies has to be addressed.
