New research suggests that nanoparticles may be effectively used to interfere with the expression of specific genes and provide targeted disease treatment.  A study published in Nature Nanotechnology reports on promising developments involving the use of nanoparticles in gene therapy.   Researchers at Massachusetts Institute of Technology, Alnylam Pharmaceuticals, Harvard Medical School, University of Colorado, and Technion Israel Institute of Technology published the study titled In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight. Nature Nanotechnology 9, 648–655 (2014).

According to the authors, the study shows that “polymeric nanoparticles made of low-molecular-weight polyamines and lipids can deliver siRNA to endothelial cells with high efficiency, thereby facilitating the simultaneous silencing of multiple endothelial genes in vivo.” 

Endothelial cells line the heart, blood vessels, lymphatics, and serous cavities of the human body, and accordingly dysfunctional endothelial cells are implicated in many different diseases.  Small interfering RNAs (siRNA) are used in gene therapy to interfere with the expression of specific genes.  Efficient delivery of siRNA to endothelial cells using nanoparticles is a potentially promising development in the continuous search for effective, targeted, treatment of many different diseases.

The application of cell-specific therapy involving targeted interference of specific gene expression had enormous potential for the future treatment of disease.  This, and other similar research, warrants substantial attention not only for its medical and scientific implications, but also for the legal and regulatory ramifications as well.