Nanoscale Small Interfering RNA Delivery Systems For Personalized Cancer Therapy
- 1. Developmental Therapeutics Program, Children’s Center for Cancer and Blood Diseases, Children’s Hospital Los Angeles MS#160, Los Angeles, CA, USA, Division of Hematology-Oncology, Department of Pediatrics, Children’s Center for Cancer and Blood Diseases, University of Southern California Keck School of Medicine and, Los Angeles, CA 90027-0367, USA
- 2. Developmental Therapeutics Program, Children’s Center for Cancer and Blood Diseases, Children’s Hospital Los Angeles MS#160, Los Angeles, CA, USA
Description
Nanoparticles represent particularly attractive delivery systems for small interfering RNA (siRNA) and may provide the foundation for rational design and formulation of RNAi-triggering nanomedicines. siRNA can be delivered with a therapeutic intent using lipid-based delivery platforms such as stable nucleic acid lipid particles (SNALP) with a lipid bilayer containing cationic as well as fusogenic lipids and a diffusible PEG-lipid coat, polymers, cationic complexes, recombinant fusion proteins, conjugates, or polyconjugates. Several investigators have reported preclinical or early clinical proof of concept studies demonstrating that systemic delivery of siRNA nanoparticles targeting specific gene transcripts can elicit biologic responses. Therapeutic nanoparticles containing siRNA targeting specific genes that contribute
to the aggressiveness and/or radiochemotherapy resistance of cancer cells may facilitate a paradigm shift in modern cancer therapy.
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IJNST-2167-8685-01-102.pdf
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