Nano containers developed at Johns Hopkins could deliver medical compounds to targeted cells

Johns Hopkins researchers 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, the university is reporting.
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, according to a university article.

A report on their work appears in Science Advances.

Most medicines spread throughout the body in an indiscriminate way and don't target a specific cell, Jordan Green, Ph.D., leader of the research team, said in the article. “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,” Green said in the article. Green is a BMES member.

Green is a 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.

According to the article, 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.

Read the full article HERE.