Biomedical Engineering Reference
In-Depth Information
in the targeted LCP showed significant improvement over those in the
untreated group. The anti-metastasis activity of the combined siRNA
was highly dependent on the LCP formulation and the sequence of
the siRNA.
155
3.5
Conclusion
There have been extensive studies on gene therapy, especially RNAi,
as a promising therapeutic modality in cancer and other diseases.
The development of a safe and effective carrier system is the key
element for successful gene therapy. An ideal carrier system has
to show the following features: high encapsulation efficiency,
stability in the circulation, specificity to the target, fine control of
loading and release of the gene, prolonged gene expression, and low
immunotoxicity. Among many other approaches in developing non-
viral carriers, the cationic lipid membrane/core NPs have shown
special promise because of their ability to form stable particles
with different negatively charged nucleic acids and the relative
ease of fabricating both the core and the surface functionalized
with a targeting moiety. However, both the ability to overcome
cellular barriers (e.g., avoiding the RES uptake, cargo release from
the endosome, and intracellular trafficking of the cargo DNA to the
nucleus) and a relatively low transfection efficiency compared to
the viral vectors still remain as major challenges for future
development. Researchers may eventually be able to overcome
these challenges by using rapidly advancing nanotechnology to
maximize the clinical significance of a new platform of carrier
systems.
Acknowledgment
The original work in this laboratory was supported by NIH grants
CA129835, CA129421, CA149363, CA151652, and CA151455. We
thank Bethany DiPrete for editing the manuscript.
References
1. Pathak, A., S. Patnaik, and K.C. Gupta, Recent trends in non-viral vector-
mediated gene delivery.
Biotechnol J
, 2009.
4
(11): pp. 1559-1572.
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