Nanocontainer Ships Titan-Size Gene Therapies and Drugs Into Cells
Johns Hopkins University School of Medicine via phys.org / Dec. 6, 2019Scientists at Johns Hopkins Medicine report they have created a tiny, nanosize container that can slip inside cells and deliver protein-based medicines and gene therapies of any size—even hefty ones attached to the gene-editing tool called CRISPR. If their creation—constructed of a biodegradable polymer—passes more laboratory testing, it could offer a way to efficiently ferry larger medical compounds into specifically selected target cells.
A report on their work appears in the Dec. 6 issue of Science Advances.
“Most medicines spread throughout the body in an indiscriminate way and don’t target a specific cell,” says biomedical engineer Jordan Green, Ph.D., leader of the research team. “Some medicines, such as antibodies, latch on to targets on the cell’s surface receptors, but we don’t have good systems for delivering biological medicines straight to the inside of a cell, which is where therapies would have the best chance at working properly and with fewer side effects.”
OK, stand by for the credentials of a guy who did not waste his time in college taking ridiculous courses:
Many academic and commercial scientists have long sought better transit systems for therapies, says Green, professor of biomedical engineering, ophthalmology, oncology, neurosurgery, materials science and engineering, and chemical and biomolecular engineering at the Johns Hopkins University School of Medicine, and a member of the Bloomberg~Kimmel Institute for Cancer Immunotherapy at Johns Hopkins.
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Some commercially available techniques use stripped down forms of viruses—known for their ability to “infect” cells directly—to deliver therapies, although the noninfectious versions of these delivery systems can unleash an unwanted immune system response.
Other therapies aimed at diseased blood cells, for example, are more cumbersome, requiring patients’ blood to be removed, then zapped with an electric current that opens pores in the cell membrane to gain entry.
The nanosize container that Green and his team developed at Johns Hopkins borrows an idea from the properties of viruses, many of which are nearly spherical in shape and carry both negative and positive charges.
With a more neutral overall charge, viruses can get close to cells. That’s not the case with many biological medicines, which consist of highly charged, large proteins and nucleic acids that tend to repel off cells.
I’d rather imagine Raquel Welch rappelling off cells in her white scuba suit, but that’s why I’m not a Professor (who totally could’ve scored with Ginger or Mary Ann if he put in any effort).
