Nucleic-acid-based molecules (DNA, RNA) have emerged in recent years as promising drugs for treating a range of genetic diseases, including cancer, diabetes, cystic fibrosis, hemophilia, and infectious, cardiovascular, inflammatory, and neurodegenerative diseases. Currently, the use of nucleic-acid drugs is limited by several factors, including inefficient delivery to target tissues and cells.
“Drug delivery is a really substantial hurdle that needs to be overcome,” says James Dahlman, a former graduate student at MIT and now an assistant professor in the Wallace H. Coulter Dept. of Biomedical Engineering at Georgia Tech and Emory Univ. “Regardless of their biological mechanisms of action, all genetic therapies need safe and specific drug delivery to the tissue you want to target.”
For the past 20 years, scientists have been developing nanoparticles as drug-delivery vehicles. The testing has been slow-going, however, because there are so many possible combinations of materials available (thousands of nanoparticles with distinct chemical structures and properties) and many nanoparticles must be tested in vitro in cell culture before a small number can be evaluated in vivo in animals.
Scientists at the Univ. of Florida, Georgia Tech, and...
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